Immediately after the FDA said it found elevated levels of the carcinogenic ingredient N-nitrosodimethylamine (NDMA) in certain extended release (ER) metformin products, the first 2 major recalls are underway.

Both Apotex Corp and Amneal Pharmaceuticals have issued a voluntary recall of metformin hydrochloride extended-release tablets, and FDA said three additional companies will recall their ER metformin.

Apotex Corp is voluntarily recalling all lots of metformin hydrochloride extended-release tablets, USP 500 mg, within expiry to retail level. However, Apotex stopped selling this product in the U.S. in February 2019, and there remains only limited product on the market. To date, Apotex has not received any reports of adverse events related to use of that product, it said.

Amneal is voluntarily recalling all lots of metformin hydrochloride extended-release tablets, USP, 500 mg and 750 mg, within expiry to the retail levels. The affected products were distributed nationwide in the US directly to wholesalers, distributors, retailers, and repackagers.

Pharma maker recalls drug over undeclared anti-hypertensive drug

The agency’s testing found that “certain extended release metformin products contain NDMA above the acceptable level,” FDA said.

“FDA is contacting companies with ER metformin above the acceptable level and will continue to take quick and appropriate action when needed to protect American consumers. We are committed to providing timely updates on our investigations and any recalls,” FDA said.

The FDA said that patients should not stop taking their prescription medications without first talking to their healthcare providers.

Pharma maker pulls weight loss drug over FDA concerns

In early February, the agency posted the results of its laboratory analysis showing NDMA levels in some samples of the metformin products it tested. Overall, the levels of NDMA found in metformin ranged from “not detectable to low levels” at the time.

According to the agency, no sample of metformin that was tested by the FDA exceeded the acceptable daily intake of NDMA.

In December, regulatory agencies outside of the United States recalled some metformin drugs due to low levels of the impurity, prompting FDA officials to test samples of metformin sold in the US.

More ranitidine recalled, plus another big drug recall

Articles

Two metformin products recalled, more on the way

FDA cleared the only drug in the U.S. to treat severe malaria in adults and children. There has been no FDA-approved drug to treat severe malaria since the marketing of quinine was discontinued by the manufacturer in March 2019, the agency said in a 

FDA okayed IV artesunate (Amivas), which should always be followed by a complete treatment course of an appropriate oral antimalarial regimen.

Previously, IV artesunate was only available to patients through the FDA’s Expanded Access program, which allowed the CDC  to provide IV artesunate to U.S. patients with severe malaria and to patients with uncomplicated malaria who are unable to take oral medications under an investigational new drug (IND) protocol.

FDA clears new antigen, saliva tests for COVID-19

“This approval will now give patients more access to a lifesaving drug,” said John Farley, MD, acting director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research. “Furthermore, the risk of developing severe malaria emphasizes the importance of taking medications to prevent malaria and using mosquito avoidance measures when traveling to malaria-endemic areas.”

According to the CDC, approximately 2,000 cases of malaria are diagnosed in the U.S. each year, and around 300 of those infected have severe disease. Most people diagnosed with malaria in the United States acquire it during travel to countries with malaria.

The safety and efficacy of IV artesunate was primarily evaluated in a randomized controlled trial in Asia (Trial 1) and a supportive published randomized controlled trial in Africa (Trial 2).

In both trials, the number of patients treated with artesunate who died in the hospital was significantly lower than the number who died in the control group treated with quinine.

In Trial 1, the most common adverse reactions in patients with malaria treated with IV artesunate were acute renal failure requiring dialysis, hemoglobinuria and jaundice. The safety profile in Trial 2 was generally similar to Trial 1.

IV artesunate is the only drug available in the U.S. to treat the disease.

FDA approves med for severe malaria

MasterPharm is voluntarily recalling 1 lot of Finasteride Plus 1.25 mg. capsules to the consumer level.

The capsules undeclared minoxidil, an antihypertensive drug, at levels greater than those found in FDA approved products, MasterPharm said in a 

. MasterPharm has received 33 reports of increased heart rate, retention of water, dizziness and low blood pressure.

Consumption of undeclared minoxidil would be expected to result in low blood pressure, rapid heartbeat, and salt and water retention causing swelling. Consequently, patients may be at risk for developing heart failure or other heart damage,” MasterPharm said.

Excess fluid between the heart and the sac surrounding the heart has also been reported in association with minoxidil use.

Finasteride Plus is a compounded drug for hair loss and is packaged in orange prescription bottles containing 30 capsules or blue prescription bottles containing 90 capsules.

Epilepsy, bipolar drug recalled due to contamination

The product was distributed nationwide on a patient-specific, prescription basis only, MasterPharm said. The affected Finasteride Plus 1.25 mg. lot number is 02-27-2020:04@11, and it has a Beyond Use Date of August 25, 2020.

Consumers should contact their physician or healthcare provider if they have experienced any problems related to taking the product.

Consumers that have the Finasteride Plus lot that is being recalled should stop using it and return it to MasterPharm via self-addressed packaging, which has been sent to all customers.

Consumers with questions can contact MasterPharm at (866) 630-5600 or 

FDA authorized the first stand-alone, at-home COVID-19 test, plus it warned more companies over COVID-19 claims and fast-tracked a promising vaccine in the top news articles this week. Here is a quick roundup:

emergency use authorization (EUA) to Everlywell, Inc.

 for its Everlywell COVID-19 Test Home Collection Kit, which is authorized to be used by individuals at home who have been screened using an online questionnaire reviewed by a health care provider.

“This allows an individual to self-collect a nasal sample at home using Everlywell’s authorized kit. The FDA has also authorized two COVID-19 diagnostic tests, performed at specific laboratories, for use with samples collected using the Everlywell COVID-19 Test Home Collection Kit,” the agency said in a 

“The authorization of a COVID-19 at-home collection kit that can be used with multiple tests at multiple labs not only provides increased patient access to tests, but also protects others from potential exposure,” said Jeffrey Shuren, MD, director of the FDA’s Center for Devices and Radiological Health. “Today’s action is also another great example of public-private partnerships in which data from a privately funded study was used by industry to support an EUA request, saving precious time as we continue our fight against this pandemic.”  

2. FDA issued warning letters to two companies for selling fraudulent COVID-19 products. It warned 

, which claims its CBD products may help with COVID-19.

FDA: Fraudulent COVID-19 medical products abound

“We are getting a lot of questions about whether CBD (Hemp Extract) can help with the Coronavirus (COVID-19),” Noetic said on its web site. “The short answer is YES, it certainly can help. Now, let’s be clear, it is not seen as a cure, but CBD has properties (anti-bacterial, anti-viral, and anti-inflammatory) that can aid with symptoms and help a person stay healthy to fight off illness.”

, which claims that its kratom products are safe and/or effective for the prevention and treatment of COVID-19. “Kratom contains a special compound known as chloroquine or Cq, that research shows is powerfully combative against the coronavirus,” the company said on its web site.

3. FDA granted "fast track" designation to Moderna’s experimental COVID-19 vaccine, Reuters 

. The pharma maker expects to start a late-stage study of the vaccine in early summer and says there is potential for a marketing application approval in 2021, Reuters said.

Moderna announced positive interim clinical data of the vaccine, mRNA-1273, from its Phase 1 study led by the National Institute of Allergy and Infectious Diseases (NIAID).

“These interim Phase 1 data, while early, demonstrate that vaccination with mRNA-1273 elicits an immune response of the magnitude caused by natural infection starting with a dose as low as 25 µg,” said Tal Zaks, MD, PhD, chief medical officer at Moderna, in a 

FDA warns CBD companies making medical claims

FDA authorizes first stand-alone, at-home test, plus other top COVID-19 news stories.

Top 3 COVID-19 stories this week

Late last week, FDA approved the first antigen test for COVID-19 as well as the first diagnostic test with the option of using home-collected saliva samples.

emergency use authorization (EUA) for a COVID-19 antigen test

, a new category of tests. The Sofira 2 SARS Antigen FIA (Quidel Corporation) is authorized for use in high and moderate complexity laboratories certified by 

Clinical Laboratory Improvement Amendments (CLIA)

, as well as for point-of-care testing by facilities operating under a CLIA Certificate of Waiver.

The antigen tests quickly detect fragments of proteins found on or within the virus by testing samples collected from the nasal cavity using swabs, FDA said in a 

FDA clears first home COVID-19 test, plus other top news

To date, FDA has approved polymerase chain reaction (PCR) tests, a molecular diagnostic testing technique that detects the genetic material from the virus and can help diagnose an active COVID-19 infection. It has also cleared serological tests that look for antibodies to the virus.

PCR tests can be incredibly accurate, but running the tests and analyzing the results can take time.

"One of the main advantages of an antigen test is the speed of the test, which can provide results in minutes. However, antigen tests may not detect all active infections, as they do not work the same way as a PCR test,” the FDA said.

While antigen tests are very specific for the virus, they are not as sensitive as molecular PCR tests.

“This means that positive results from antigen tests are highly accurate, but there is a higher chance of false negatives, so negative results do not rule out infection,” the FDA said. “With this in mind, negative results from an antigen test may need to be confirmed with a PCR test prior to making treatment decisions or to prevent the possible spread of the virus due to a false negative.”

FDA authorizes treatment, rapid test for COVID-19

Antigen tests can generally be produced at a lower cost than PCR tests and, once multiple manufacturers enter the market, can potentially scale to test millions of Americans per day due to their simpler design, “helping our country better identify infection rates closer to real time,” FDA said.

 (EUA) to Rutgers Clinical Genomics Laboratory for its COVID-19 laboratory developed test (LDT), to permit testing of samples self-collected by patients at home using the Spectrum Solutions LLC SDNA-1000 Saliva Collection Device.

“Authorizing additional diagnostic tests with the option of at-home sample collection will continue to increase patient access to testing for COVID-19. This provides an additional option for the easy, safe and convenient collection of samples required for testing without traveling to a doctor’s office, hospital or testing site,” according to FDA Commissioner Stephen M. Hahn, MD, in a 

“The Rutgers Clinical Genomics Laboratory test is currently the only authorized COVID-19 diagnostic test that uses saliva samples to test for SARS-CoV-2, the strain of coronavirus that causes COVID-19. The test remains prescription only,” the FDA said.

The Rutgers test allows testing of a saliva sample collected from the patient using a designated self-collection kit. Once patients collect their saliva sample, they return it to the Rutgers Clinical Genomics Laboratory in a sealed package for testing.

However, FDA’s authorization is limited to testing performed at the Rutgers Clinical Genomics Laboratory using its molecular LDT COVID-19 authorized test for saliva specimens collected using the Spectrum Solutions LLC SDNA-1000 Saliva Collection Device.

Antigen tests, a new category, have a major advantage and disadvantage over COVID-19 tests already approved.

FDA has warned more than 40 companies that have made fraudulent medical claims relating to COVID-19.

 to companies making fake COVID-19 claims, including to a seller of fraudulent chlorine dioxide products, equivalent to industrial bleach, as a treatment for COVID-19.

FDA, FTC warn suppliers of fraudulent COVID-19 products

The chorine dioxide products are frequently referred to as “Miracle Mineral Solution” or “MMS.”

"After the seller refused to take corrective action, a federal court issued a 

 requiring the seller to immediately stop distributing its unproven and potentially dangerous product,” FDA said in a 

FDA has also discovered hundreds of fraudulent drugs, testing kits and personal protective equipment (PPE) sold online with unproven claims.

“We continue to work with online marketplaces, domain name registrars, payment processors and social media websites to remove from their platforms products that fraudulently claim to mitigate, prevent, treat, diagnose, or cure COVID-19 and to keep those products from reappearing under different names,” the agency said.

“While we seek to ensure access to critical medical products, it is imperative that we continue our efforts to find and prevent the sale and distribution of products that may be harmful to the public health. Americans can rest assured that we’re leveraging our experience investigating, examining, and reviewing medical products, both at the border and within domestic commerce, to help ensure that the critical resources reaching the frontlines in the battle against COVID-19 are appropriate,” said FDA Associate Commissioner for Regulatory Affairs Judy McMeekin, PharmD.

Drugmaker halts access to COVID drug as FDA approves novel test

FDA is also keeping unproven products out of the country. It recently intercepted and investigated a 

 of mislabeled COVID-19 “treatment kits” offered for import.

“As a result, special agents with the FDA’s Office of Criminal Investigations, with the help of domestic and international law enforcement counterparts in the United Kingdom, led the Department of Justice to bring a criminal complaint against a British man who sought to profit from this pandemic and jeopardize public health,” the FDA said.

Numerous PPEs and testing kits also feature unproven coronavirus claims.

FDA cleared a new subcutaneous formulation of daratumumab for multiple myeloma patients.

Daratumumab and hyaluronidase-fihj (Darzalex Faspro, Janssen) is approved in four regimens across five indications in multiple myeloma patients, including newly diagnosed, transplant-ineligible patients as well as relapsed or refractory patients. 

New Multiple Myeloma Treatment Works By Targeting Cells

As a fixed-dose formulation, the medication can be administered over approximately three to five minutes, which is significantly less time than Darzalex (Janssen), which is given intravenously over hours, Janssen said in a 

Net trade sales of Darzalex reached $463 million in the U.S. and $474 million in the rest of the world in the first quarter of 2020, Genmab said in a 

Merck’s Keytruda Snags Another Cancer Indication

"Since the approval of daratumumab, a robust body of evidence has established its use as a treatment for multiple myeloma in both the frontline and relapsed and refractory settings," said Saad Z. Usmani, MD, division chief of Plasma Cell Disorders at Levine Cancer Institute, in the Janssen press release. "With Darzalex Faspro, there may be fewer administration-related reactions compared to intravenous Darzalex, providing an additional treatment option that may help patients, oncologists and nursing staff.”

In fact, Janssen’s Phase 3 trial showed a nearly two-thirds reduction in systemic administration-related reactions (ARRs) for Darzalex Faspro compared to intravenous Darzalex (13% vs. 34%, respectively).

The study also demonstrated a consistent overall response rate (ORR) and pharmacokinetics and a similar safety profile compared with IV Darzalex, Janssen said.

Formulation could cause fewer administration-related reactions than IV Darzalex.

New subcutaneous multiple myeloma treatment available

The first COVID-19 vaccine candidates could be ready as early as this fall, according to vaccine developers.

AstraZeneca and the University of Oxford 

 to distribute the University’s potential recombinant adenovirus vaccine aimed at preventing COVID-19 infection from SARS-CoV-2.

FDA restricts hydroxychloroquine use, plus other COVID-19 updates

“Our hope is that, by joining forces, we can accelerate the globalization of a vaccine to combat the virus and protect people from the deadliest pandemic in a generation,” said AstraZeneca CEO Pascal Soriot in a 

The potential vaccine entered Phase I clinical trials in late April at five centers in Southern England. Data from the Phase I trial could be available next month, while advancement to late-stage trials should take place by the middle of this year, AstraZeneca and the University of Oxford said.

Similarly, BioNTech and Pfizer Inc. are moving quickly on a COVID-19 vaccine. Twelve study participants were dosed with vaccine candidate BNT162 in Germany since dosing began April 23.

“The trial is the first clinical trial of a COVID-19 vaccine candidate in Germany. Pfizer and BioNTech plan to initiate trials for BNT162 in the U.S. upon regulatory approval, which is expected shortly,” the two companies said in a 

However, Pfizer and BioNTech did not say when they expect their vaccine candidate to be ready for manufacturing.

- Amazon, Walgreens, and CVS are facing shortages of famotidine, the active ingredient in Pepcid AC and other heartburn medications after a clinical trial on the ingredient was launched.

Northwell Health in New Hyde Park, NY, is testing famotidine intravenously, at nine times the typical dose.

“Unlike other drugs the 23-hospital system is testing, including Regeneron’s sarilumab and Gilead Sciences’s remdesivir, Northwell kept the famotidine study under wraps to secure a research stockpile before other hospitals, or even the federal government, started to buy it,” Science magazine 

On Amazon, Pepcid AC and all three of its generic versions were unavailable or out of stock as of April 27, Business Insider 

. Walgreens also showed out-of-stock notices at most locations across the U.S.

- FDA issued Emergency Use Authorization for Roche’s Elecsys® Anti-SARS-CoV-2 antibody test. The test is designed to help determine if a patient has been exposed to the SARS-CoV-2 virus and if the patient has developed antibodies against SARS-CoV-2, the pharma maker said in a 

“Roche has already started shipping the new antibody test to leading laboratories globally and will ramp up production capacity to high double-digit millions per month to serve healthcare systems in countries accepting the CE mark2 as well as the U.S.,” Roche said.

With extensive global manufacturing capabilities, Roche will be able to deliver high double-digit millions of tests per month, it added. “Hospitals and reference laboratories can run the test on Roche’s cobas e analysers, which are widely available around the world.”

Another RA drug starts testing to treat COVID-19

Popular heartburn medication now in shortage due to COVID-19 test.

COVID-19 vaccines could be ready this year, plus other top news

FDA warned about serious heart rhythm problems in COVID-19 patients treated with hydroxychloroquine or chloroquine, often in combination with azithromycin.

The drugs should only be used to treat COVID-19 patients who are hospitalized or are in clinical trials, so doctors and screen and supervise them, the agency said in a new 

“The FDA is aware of reports of serious heart rhythm problems in patients with COVID-19 treated with hydroxychloroquine or chloroquine, often in combination with azithromycin and other QT prolonging medicines. We are also aware of increased use of these medicines through outpatient prescriptions,” the agency said in a 

. “Therefore, we would like to remind health care professionals and patients of the known risks associated with both hydroxychloroquine and chloroquine.”

Hydroxychloroquine and chloroquine have not been shown to be safe and effective for treating or preventing COVID-19, the agency said. “They are being studied in clinical trials for COVID-19, and we authorized their temporary use during the COVID-19 pandemic for treatment of the virus in hospitalized patients when clinical trials are not available, or participation is not feasible,through an 

Hydroxychloroquine and chloroquine can cause abnormal heart rhythms such as QT interval prolongation and a dangerously rapid heart rate called ventricular tachycardia. 

“These risks may increase when these medicines are combined with other medicines known to prolong the QT interval, including the antibiotic azithromycin, which is also being used in some COVID-19 patients without FDA approval for this condition. Patients who also have other health issues such as heart and kidney disease are likely to be at increased risk of these heart problems when receiving these medicines,” FDA said in the press release.

Trump, Fauci disagree on the effectiveness of hydroxychloroquine

1. Gilead Science’s clinical trial of the rheumatoid arthritis drug remdesivir was not effective in treating COVID-19, according to a draft of a Chinese study that was inadvertently posted to the World Health Organization (WHO) web site early, TIME 

Remdesivir was “not associated with a difference in time to clinical improvement,” according to STAT, and there was almost no difference in the fatality rate of patients given remdesivir compared to those who received the placebo, TIME said.

However, the study had to be terminated early due to low enrollment. “We believe the post included inappropriate characterizations of the study. The study was terminated early due to low enrollment and, as a result, it was underpowered to enable statistically meaningful conclusions,” Merdad Parsey, MD, chief medical officer at Gilead Sciences, told TIME. “As such, the study results are inconclusive, though trends in the data suggest a potential benefit for remdesivir, particularly among patients treated early in the disease.”

2. There is no evidence that people who have recovered from COVID-19 are protected from a second infection, 

. Still, most studies suggest that people who have recovered from the infection have antibodies to fight the virus, WHO said.

WHO advised against the use of “immunity passports,” which have been suggested in some countries as a way to allow people to travel or return to work, NBC News said.

There is not enough evidence about the effectiveness of antibody-mediated immunity to guarantee the accuracy of such passports, WHO said.

Agency says the anti-malaria drug can cause heart rhythm problems.

FDA is warning two companies for illegally selling cannabidiol (CBD) products that they claim can help treat diseases and opioid addiction.

 for distributing injectable CBD products and an injectable curcumin product, which are marketed for serious diseases and as an alternative to opioids.

“VISION: OPIOID-FREE FUTURE . . . Our goal is to supply the world with pharmaceutical grade, all natural products containing cannabidiol and other natural compounds. We believe strongly that pharmaceutical grade hemp oil will drastically reduce the need for opioid-based pain relief and eliminate the global opioid epidemic by providing a safe and natural alternative,” BIOTA said on its web site, FDA said in a 

Homero Corp DBA Natures CBD Oil Distribution

 includes claims such as “Natures Pure CBD has had success in alleviating severe withdrawal symptoms associated with opiate dependency,” on its web site. Homero is an own label distributor for CBD products as well as a retailer for Green Roads CBD products.

“The opioid crisis continues to be a serious problem in the US, and we will continue to crack down on companies that attempt to benefit from selling products with unfounded treatment claims,” said FDA Principal Deputy Commissioner Amy Abernethy, MD, PhD.

FDA says CBD not recognized as safe In food, warns manufacturers

“CBD has not been shown to treat opioid addiction. Opioid addiction is a real problem in our country, and those who are addicted need to seek out proper treatment from a health care provider,” Abernethy added. “There are many unanswered questions about the science, safety, effectiveness and quality of unapproved products containing CBD, and we will continue to work to protect the health and safety of American consumers from products that are being marketed in violation of the law.”

FDA has not approved any CBD products other than 

 to treat rare, severe forms of epilepsy.

“Unlike drugs approved by the FDA, there has been no FDA evaluation of whether these unapproved products are effective for their intended use, what the proper dosage might be, how they could interact with FDA-approved drugs, or whether they have dangerous side effects or other safety concerns,” the agency said.

The agency continues to be concerned that some people wrongly think that the myriad of CBD products on the market have been evaluated by the FDA and determined to be safe, or that using CBD “can’t hurt,” FDA said. “The FDA remains focused on educating the public about the number of questions that remain regarding CBD’s safety. There may be risks that need to be considered before using CBD products outside of the monitored setting of a prescription from your health care provider.”

 on its work related to CBD products with a focus on protecting public health and providing market clarity.

Suppliers claim CBD can help alleviate opioid addiction, cure diseases.

 that the US government supply of remdesivir for hospitalized COVID-19 patients could run out by the end of June, that is not the case.

The Department of Health and Human Services agreed to secure large supplies of the drug from Gilead Sciences through September, allowing American hospitals to purchase remdesivir in amounts allocated by HHS and state health departments.

“To the extent possible, we want to ensure that any American patient who needs remdesivir can get it. The Trump Administration is doing everything in our power to learn more about life-saving therapeutics for COVID-19 and secure access to these options for the American people,” HHS Secretary Alex Azar said in a 

HHS has secured more than 500,000 treatment courses of the drug for American hospitals through September.

FDA pulls hydroxychloroquine for COVID-19, warns about other drug interaction

Hospitals will pay no more than Gilead’s Wholesale Acquisition Price (WAC) — approximately $3,200 per treatment course, HHS said.

However, remdesivir could be priced up to $5,080 per course based on benefits shown in COVID-19 patients, the Institute for Clinical and Economic Review (ICER) said in a 

HHS allocates product to state and territorial health departments based on COVID-19 hospital burden, and health departments allocate it to hospitals. The delivery of the purchased remdesivir will be streamlined, going directly to the hospital, per the state’s allocation decision, rather than going first to the state health departments for subsequent delivery to hospitals, HHS said.

Price of the drug to treat hospitalized patients revealed.

U.S. government secures remdesivir supply for COVID-19 patients

In the past week, FDA cleared two novel cancer treatments — one for breast cancer and the other for colorectal cancer.

FDA cleared a combination of pertuzumab, trastuzumab and hyaluronidase–zzxf (Phesgo, Genentech) for injection under the skin to treat adult patients with HER2-positive breast cancer that has spread to other parts of the body. Phesgo also treats adult patients with early HER2-positive breast cancer.

Pharma maker seeks fast approval of lymphoma treatment

“Currently, most patients with HER2-positive breast cancer receive trastuzumab and pertuzumab at infusion centers. With a new administration route, Phesgo offers an out-patient option for patients to receive trastuzumab and pertuzumab,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, in a 

“As part of the FDA’s ongoing commitment to address the novel coronavirus pandemic, we continue to keep a strong focus on patients with cancer who constitute a vulnerable population at risk of contracting the disease,” Pazdur added. “At this critical time, we continue to expedite oncology product development. This application was approved about four months ahead of the FDA goal date.”

The FDA’s approval was based on the results of a non-inferiority study in patients with HER2-positive early breast cancer, which demonstrated Phesgo had comparable efficacy and safety as IV pertuzumab and IV trastuzumab, except for administration-related reactions, which were higher with Phesgo due to the subcutaneous route of administration.

Prescribing information for Phesgo includes a boxed warning to advise health care professionals and patients about the risk of potential heart failure, fetal harm and lung toxicity. Health care professionals should use similar monitoring parameters as those used with IV pertuzumab and IV trastuzumab, FDA said.

FDA also approved pembrolizumab (Keytruda, Merck & Co.) for IV injection for the first-line treatment of patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer.

“This marks the first immunotherapy approved for this patient population as a first-line treatment and which is administered to patients without also giving chemotherapy,” FDA said in a 

“Metastatic colorectal cancer is a serious and life-threatening disease with a poor prognosis. Available current therapy with chemotherapy combinations and other biologics are associated with substantial toxicity. Having a non-chemotherapy option available for selected patients is a noteworthy paradigm shift in treatment,” Pazdur said.

FDA’s approval was based on the results of one multicenter, international, open-label, active-controlled, randomized trial that compared Keytruda with chemotherapy treatment in 307 patients with MSI-H or dMMR metastatic colorectal cancer. The study demonstrated a statistically significant improvement in progression-free survival (PFS): median PFS was 16.5 months in the Keytruda group and 8.2 months in the standard of care group, FDA said.

“Longer-term analysis is needed to assess for an effect on survival,” the agency added.

Colorectal cancer drug is the first immunotherapy administered as a first-line treatment and without chemotherapy.

FDA okays novel breast, colorectal cancer treatments

While there are numerous drugs in the pipeline, pharmacy benefit manager OptumRx singled out three top medications expected to have the most impact on payers on patients in the second half of 2020.

Pipeline Insights Report: 3 Drugs to Watch

, OptumRx highlights treatments for breast cancer, spinal muscular atrophy, and Duchenne muscular dystrophy. Here are the top 3 drugs to watch in the second half of this year:

1. Sacituzumab govitecan-hziy (Trodelvy, Immunomedics) was approved in late April to treat patients with metastatic triple-negative breast cancer (TNBC) who have received at least two prior therapies for metastatic disease.

TNBC is an aggressive breast cancer that accounts for up to 20% of all breast cancer cases.

Trodelvy is a novel antibody-drug conjugate (ADC), which combines both biologically engineered antibodies andtraditional chemotherapy drugs, OptumRx said. It is administered intravenously on a weekly basis over a 21-day cycle.

“Trodelvy is the first ADC targeted therapy for TNBC. There is a high unmet need for treatments in this subtype of breast cancer because of the size of the overall breast cancer population and the aggressive nature of TNBC,” OptumRx said.

In fact, analysts estimate that Trodelvy could reach $1.44 billion in global sales in 2024 global sales, initially as a third-line treatment for late-stage TNBC. The drug is also being evaluated in earlier settings and other cancers.

2. The investigational drug risdiplam (F. Hoffmann-La Roche, PTC Therapeutics, SMA Foundation) would be the first oral therapy to treat spinal muscular atrophy (SMA).

SMA is a rare group of severe neuromuscular disorders that leads to progressive muscle weakness and atrophy. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies, OptumRx said.

Express Scripts report: Top drug classes driving spending growth

“There is an unmet need for treatments for SMA, and especially for an all-oral option. There are currently only two FDA- approved drugs for SMA,” OptumRx said.

Nusinersen (Spinraza, Biogen) requires repeated, invasive injections into the cerebrospinal fluid in the spinal cord. The other alternative isonasemnogene abeparvovec (Zolgensma, AveXis) which is a one-time IV-infused gene therapy.

FDA is expected to approve the medication on or before August 24, 2020, according to OptumRx.

“While Spinraza does require invasive spinal injections, it has been widely used in SMA patients since its market entry. Practically speaking, the competitive advantage for risdiplam will rest mainly in its oral route of administration, and perhaps, a lower cost,” OptumRx said.

3. Viltolarsen (Nippon Shinyaku) is in development to treat specific forms of Duchenne muscular dystrophy (DMD). DMD is a rare genetic disorder that affects young boys.

There is a significant unmet need for treatments for DMD since it is associated with substantial sickness and death,” OptumRx said.

If approved, viltolarsen would be the third exon-skipping drug for DMD, and the second specifically for the 8% of patients with a specific mutation.

Golodirsen (Vyondys 53TM, Sarepta Therapeutics) is also approved for the mutation, but there is limited efficacy data and unknown clinical benefit with its use, OptumRx said.

The FDA is expected to make a decision on whether or not to approve viltolarsen in the third quarter of this year.

Read more: 

These are the top 3 drugs recently approved — or soon to be approved by FDA — to watch in 2020.

Top 3 drugs to watch in second half 2020

TG Therapeutics completed the rolling submission of a New Drug Application (NDA) to FDA, requesting accelerated approval of umbralisib to treat certain types of lymphoma.

The investigational once-daily, oral, dual inhibitor of PI3K-delta and CK1-epsilon will treat patients with previously treated marginal zone lymphoma (MZL) and follicular lymphoma (FL).

FDA previously granted umbralisib breakthrough therapy designation (BTD) for MZL and orphan drug designation (ODD) for MZL and FL.

"The completion of this NDA submission marks an important milestone in bringing us one step closer to providing umbralisib as a potential treatment option for patients with relapsed/refractory MZL and FL,” said Michael S. Weiss, TG Therapeutics’ executive chairman and CEO in a 

TG Therapeutics is evaluating umbralisib in the UNITY-NHL trial , a multicenter, open-label Phase 2b trial.

In February 2019, the company announced that the primary endpoint of overall response rate (ORR) as determined by Independent Review Committee (IRC) was met for all treated MZL patients. The results met the company’s target guidance of 40-50% ORR.

Investigational treatment will target marginal zone lymphoma (MZL) and follicular lymphoma (FL). 