While approval of several drugs have been delayed due to COVID-19, FDA has still cleared more than 30 new drugs this year — ahead of its average rate, a new OptumRx report says.

Here are the top 5 drugs in the pipeline for 2020 — either approved recently or expected to garner FDA clearance this year — according to OptumRx’s most recent Drug Pipeline Insight Report.

 Valoctocogene roxaparvovec (Roctavian, BioMarin) to treat hemophilia A in adults. While an FDA decision on whether or not the approve the medication was expected by August 21, the agency requested additional data from BioMarin.

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“Existing treatments for this condition are extremely expensive, costing up to $500,000 per year. Valoctocogene roxaparvovec would offer a one-time gene therapy that could replace the need for these treatments in some patients,” OptumRx wrote.

The projected wholesale acquisition cost (WAC) for valoctocogene roxaparvovec is expected to be in the range of $2 million to $3 million per one-time dose.

 Filgotinib (Jyseleca, Gilead Sciences) to treat moderate-to-severe rheumatoid arthritis (RA). The drug has yet to obtain FDA approval.

“Just one day prior the scheduled decision date, the FDA announced that it will require additional data from two ongoing safety studies of the drug. Relatedly, the FDA has expressed concerns regarding the overall benefit/risk profile of filgotinib at the 200 mg. dose used in trials,” OptumRx wrote. “Industry analysts speculate that if these concerns cannot be satisfied, the entire development program may be dropped by the manufacturer.”

 Brexucabtagene autoleucel (Tecartus, Kite, a Gilead company) treats adult patients with relapsed or refractory mantle cell lymphoma (MCL), a rare and aggressive form of non-Hodgkin’s lymphoma (NHL). It was approved in late July.

Notably, Tecartus is a chimeric antigen receptor T-cell (CAR T) therapy, which leverages and strengthens the power of a patient’s immune system to attack cancer cells.

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“Tecartus becomes the first CAR T-cell therapy for MCL, offering an additional treatment option for patients with relapsed or refractory MCL that only requires a one-time dose administered by IV infusion,” OptumRx wrote.

In trials with a median follow-up period of 12.3 months, 93% of patients responded to a single infusion of Tecartus, including 67% of patients who achieved a complete response.

 Subcutaneous ofatumumab (Kesimpta, Novartis), approved in late August, treats relapsing forms of multiple sclerosis (MS). Ofatumumab is currently available in intravenous (IV) form as Arzerra, which is FDA approved for the treatment of chronic lymphocytic leukemia.

Kesimpta will be entering a crowded MS marketplace, competing not only with Aubagio, but also with other well established oral and injectable products with different mechanisms of action, OptumRx said.

“Analysts say that being self-administered by subcutaneous injection would give Kesimpta a key advantage,” OptumRx said.

 Tafasitamab-cxix (Monjuvi, MorphoSys AG), approved in late July, is the first FDA-approved, second-line regimen for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

Monjuvi is to be used in combination with lenalidomide (Revlimid). It will be competing with polatuzumab vedotin (Polivy), which was approved in June 2019 for a similar indication.

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Some of the top medications were approved this summer, while others are awaiting FDA approval.

Top 5 drugs in the pipeline

In the second recall of thyroid medications this month, Acella Pharmaceuticals is recalling one lot of its NP Thyroid due to sub-potentcy.

 of all lots of its Nature-Throid and WP Thyroid, also due to sub-potency.

The RLC Labs recall applies to 483 lots of the pharma maker’s Nature-Throid and WP Thyroid in all strengths, all counts of product within current expiry to the consumer level.

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FDA testing of samples from six lots found them to be sub-potent. The product may have as low as 87% of the labeled amount of Liothyronine (T3) or Levothyroxine(T4), RLC said in a press release.

Patients being treated for hypothyroidism who receive sub-potent Nature-Throid or WP Thyroid may experience signs and symptoms of hypothyroidism which may include: fatigue, increased sensitivity to cold, constipation, dry skin, puffy face, hair loss, slow heart rate, depression, swelling of the thyroid gland, and/or unexplained weight gain or difficulty losing weight, RLC said.

Nature-Throid and WP Thyroid (thyroid tablets, USP), packaged in 30, 60, 90, 100 and 1,000-count bottles, were distributed nationwide to retail pharmacies and healthcare professionals.

RLC Labs, Inc. is proactively notifying its wholesalers by email, mail, and phone to discontinue distribution of the product being recalled and is arranging for the return of all recalled products.

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However, patients who are currently taking the medications should not discontinue use without contacting their healthcare provider for further guidance and/or replacement prescription, the company said.

 applies to one lot of 15 mg. and one lot of 120 mg. NP Thyroid, Thyroid Tablets, USP [levothyroxine (T4) and liothyronine (T3)] , which was distributed to the consumer level.

“The products are being recalled because testing has found these lots to be sub potent. The product may have as low as 87% of the labeled amount of levothyroxine (T4),” Acella said in a press release.

Both recalls are due to sub-potency of drugs that treat hypothyroidism.

Major thyroid med recall underway

FDA granted Fast Track designation to Boehringer Ingelheim and Eli Lilly and Company to develop a new heart attack-related indication for the popular diabetes medication empagliflozin (Jardiance).

The indication will be to prevent hospitalization for heart failure and reduce the risk of mortality in patients, with and without diabetes, who have had a heart attack.

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 a new indication for semaglutide (Ozempic, Novo Nordisk) 0.5 mg. or 1 mg. injection to reduce the risk of major adverse cardiovascular events (MACE) such as heart attack, stroke, or death in adults with type 2 diabetes and known heart disease.

“Ischemic heart disease (IHD) is the leading cause of death and disability in the U.S. Myocardial infarction, or heart attack, is the deadliest acute manifestation of IHD, and treatment options are urgently needed to help improve outcomes,” said Mohamed Eid, MD, vice president of Clinical Development & Medical Affairs, Cardio-Metabolism & Respiratory Medicine at Boehringer Ingelheim, in a 

Jardiance, a once-daily tablet, is used along with diet and exercise to lower blood sugar in adults with type 2 diabetes and to reduce the risk of cardiovascular death in adults with type 2 diabetes and known cardiovascular disease.

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The pharma makers will conduct the EMPACT-MI multi-center, double-blind placebo-controlled superiority trial to investigate the effect of Jardiance on all-cause mortality and hospitalization for heart failure in adults with and without type 2 diabetes who have had an acute myocardial infarction and no history of chronic heart failure.

The results of the EMPACT-MI Phase 3 clinical trial are expected in 2023.

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The top-selling diabetes drug will join semaglutide (Ozempic), which is approved to reduce the risk of major adverse cardiovascular events.

Jardiance to get new heart attack prevention indication

FDA granted Breakthrough Therapy Designation to Toripalimab (Junshi Biosciences) to treat nasopharyngeal carcinoma. The drug is the first anti-PD-1 antibody from China to receive BTD, according to Junshi.

“The Breakthrough Therapy designation granted by FDA recognizes the significant clinical benefits Toripalimab has shown for the treatment of nasopharyngeal carcinoma,” said Ning Li, MD, CEO of Junshi Biosciences, in a 

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“Junshi prioritizes cancer types with high prevalence in China such as nasopharyngeal carcinoma, lung and liver cancer, but we found that great unmet medical needs also exist in other countries where patients are endangered by this deadly disease,” Li said. “The BTD will allow us to work with the FDA closely to bring the therapy to patients worldwide expeditiously.”

However, Junshi did not say when the drug would be available in the U.S.

The anti-PD-1 monoclonal antibody received its first approval for second-line treatment of metastatic melanoma in China in 2018.

In March, FDA granted Toripalimab in combination with axitinib in the treatment of mucosal melanoma orphan drug designation. And in May, the treatment was granted orphan drug designation by FDA to treat nasopharyngeal carcinoma.

The company has carried out more than 30 clinical studies covering more than 10 indications for the treatment.

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The treatment is the first anti-PD-1 antibody from China to receive Breakthrough therapy designation.

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FDA cleared another targeted therapy to treat non-small cell lung cancer (NSCLC), reflecting the growth in precision medicines.

Pralsetinib (Gavreto, Blueprint Medicines) is designed to treat adults with metastatic rearranged during transfection (RET) fusion-positive NSCLC as detected by an FDA-approved test.

The once-daily therapy is designed to selectively and potently inhibit RET alterations that drive many cancer types, including approximately 1% to 2% of patients with NSCLC. RET is one of seven NSCLC biomarkers that can be targeted with an FDA-approved therapy.

Blueprint Medicines and Genentech, a member of the Roche Group, will co-commercialize Gavreto in the U.S. and expect to have it available in the next week.

"Targeted therapies have dramatically improved care for patients with non-small cell lung cancer driven by oncogenes…and the approval of the selective RET inhibitor pralsetinib, or Gavreto, marks another milestone in a paradigm shift toward precision medicine," said Vivek Subbiah, MD, associate professor of Investigational Cancer Therapeutics and center medical director of the Clinical Center for Targeted Therapy at The University of Texas MD Anderson Cancer Center, and an investigator on the ARROW trial. in a Blueprint 

“This approval represents an important advance with the potential to change standards of care for patients with RET fusion-positive non-small cell lung cancer, who have historically had limited treatment options,” Subbiah added.

The Phase 1/2 ARROW clinical trial showed efficacy for Gavreto in patients with RET fusion-positive NSCLC with or without prior therapy, and regardless of RET fusion partner or central nervous system involvement, Blueprint said.

"Patients treated with GAVRETO had durable clinical responses, with a subset achieving complete responses characterized by the resolution of all target lesions, an uncommon outcome in metastatic lung cancer. We observed this activity with or without prior therapy and regardless of RET fusion partner or the presence of brain metastases,” Subbiah said.

Pharma makers expect the treatment to be available within a week.

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FDA’s approval of a novel medication to treat acute myeloid leukemia (AML) was a long time coming. 

Oral azacitidine (Onureg, Bristol Myers Squibb) took over a decade of research, including 13 pre-clinical and clinical trials, said Giovanni Caforio, MD, chairman and CEO Of Bristol Myers Squibb, in a 

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After granting Onureg’s New Drug Application Priority Review designation, FDA okayed the drug treat adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or CR with incomplete blood count recovery (CRi) following intensive induction chemotherapy and who are not able to complete intensive curative therapy.

The approval is based on results from a phase study in which treatment with Onureg resulted in a statistically significant and clinically meaningful improvement in overall survival (OS) of nearly 10 months compared to placebo.

Median OS from time of randomization was greater than two years (24.7 months) among patients who received Onureg compared to 14.8 months among patients receiving placebo, Bristol Myers Squibb said.

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“This approval should help establish continued treatment with Onureg as a standard component of AML therapy for adults who achieved first complete remission following chemotherapy and who cannot proceed to intensive curative therapy, like hematopoietic stem cell transplant,” said Andrew Wei, PhD, the study’s lead investigator and a professor and hematologist at the Alfred Hospital and adjunct professor at Monash University in Melbourne, Australia.

Wei noted that Onureg demonstrated an OS benefit in adults with AML who had achieved first complete remission and that “it has the potential to do this in a convenient manner, given its once daily oral formulation.”

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The medication boasts a statistically significant improvement in overall survival versus placebo.

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While FDA broadened the scope of its existing 

 for remdesivir (Veklury, Gilead), a recent major study showed mixed results for the COVID-19 treatment drug.

The FDA’s EUA for remdesivir now includes treatment of all hospitalized adult and pediatric patients with suspected or laboratory-confirmed COVID-19, regardless of the severity of the disease, the agency said in a 

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When FDA originally issued the EUA for remdesivir in May, the authorization was limited to patients with severe disease, defined as low blood oxygen levels, needing oxygen therapy, or more intensive breathing support, such as a mechanical ventilator.

Based on the agency’s review of all of the scientific information now available, a press release from the agency said it is “reasonable to believe Veklury may be effective for the treatment of suspected or laboratory-confirmed COVID-19 in all hospitalized adult and pediatric patients."

Gilead is working on developing an inhaled version of remdesivir. In its current formulation, the antiviral is administered intravenously.

A recent phase 3 study showed that moderately-ill patients COVID-19 patients who were on a 5-day course of remdesevir plus standard care were 65% more likely to have their condition improve compared with patients on standard care. However, for patients on a 10-day course, the chances of improvement were not statistically significant, 

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The FDA, though, pointed to a randomized, double-blind, placebo-controlled 

 (ACTT-1), funded by the National Institute of Allergy and Infectious Diseases, that evaluated how long it took for subjects to recover from COVID-19 within 29 days of being treated. The trial included 1,062 hospitalized subjects with mild, moderate, or severe COVID-19 who received remdesivir or placebo, plus standard of care. The median time to recovery from COVID-19 was 10 days for the Veklury group compared with 15 days for the placebo group. This data and other data show that remdesivir “has the potential to help even more hospitalized patients who are suffering from the effects of this devastating virus,” said FDA Commissioner Stephen M. Hahn, M.D. “We are working with drug developers to conduct randomized clinical trials to further study the safety and effectiveness of a number of 

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One study questions the effectiveness of remdesivir past day 10 while others support its expanded use.

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 the first 5-minute COVID-19 test that allows results to be read directly from a testing card, the White House said it would work with Abbott Laboratories to make 150 million tests available.

 for the first antigen test with that design, which is similar design to some pregnancy tests. The test, BinaxNow COVID-19 Ag Card, is fast and efficient for healthcare providers and patients and does not need the use of an analyzer, FDA said in a press release.

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“This means people will know if they have the virus in almost real-time. Due to its simpler design and the large number of tests the company anticipates making in the coming months, this new antigen test is an important advancement in our fight against the pandemic,” said Jeff Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health.

On August 27, the White House announced the $760 million deal with Abbott to produce 150 million tests, The Washington Post 

 that it would ship “tens of millions of tests in September, ramping production to 50 million tests a month in October.”

“This is a major development that will help our country to remain open, get Americans back to work, and kids back to school!” White House Press Secretary Kayleigh McEnany tweeted.

However, the government did not say where the tests will be distributed.

Here’s how the test works: a healthcare provider swabs the patient’s nose and twirls that sample on a test card with a testing reagent added. After waiting 15 minutes, the provider reads the results directly from the testing card. One line indicates a negative result; two lines indicate a positive result.

The pharma maker will initially produce 150 million of the 5-minute tests.

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The FDA recently approved two novel treatments for rare conditions: one for a certain mutation in Duchenne muscular dystrophy (DMD) and another for neuromyelitis optica spectrum disorder (NMOSD) for those who have a specific antibody.

The agency granted accelerated approval to viltolarsen injection (Viltepso, NS Pharma) to treat DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This is the second FDA-approved targeted treatment for patients with this type of mutation, which affects around 8% of DMD patients.

Viltepso was evaluated in two clinical studies with a total of 32 patients. In one study, dystrophin levels increased, on average, from 0.6% of normal at baseline to 5.9% of normal at week 25.

“The FDA concluded that the applicant’s data demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping,” the agency said in a 

However, a clinical benefit of the drug has not been established, FDA said. As part of the 

 process, the agency is requiring the company to conduct a clinical trial to confirm the drug’s clinical benefit.

FDA also cleared satralizumab-mwge (Enspryng, Genentech) for NMOSDin adults with a particular antibody: patients who are anti-aquaporin-4 or AQP4 antibody-positive.

Enspryng is the third approved treatment for the disorder, a rare autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord.

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“Until last year, there were no FDA-approved treatments for patients with this rare, debilitating and sometimes fatal disease. Now there are three,” said Billy Dunn, MD, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, in a 

. “Today’s approval of Enspryng highlights the FDA’s commitment to rapidly advancing safe and effective therapies for NMOSD and other neurological diseases.”

The effectiveness and safety of Enspryng was demonstrated in two, 96-week clinical studies. In the first study, Enspryng reduced the number of NMOSD relapses by 74% in patients who were anti-AQP4 positive compared to treatment with a placebo (inactive treatment).

In the second study, treatment with Enspryng reduced the number of relapses in patients who were anti-AQP4 positive by 78% compared to treatment with a placebo.

The prescribing information for Enspryng includes a warning for increased risk of infection, including serious and potentially fatal infections — such as potential reactivation of hepatitis B and tuberculosis. Other warnings and precautions for Enspryng include elevated liver enzymes, decreased neutrophil counts and hypersensitivity reactions.

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Drug for a specific Duchenne muscular dystrophy mutation is only the second drug approved for the mutation.

FDA clears 2 therapies for rare conditions

FDA accepted the new Drug Application (NDA) for umbralisib (TG Therapeutics), an investigational treatment for marginal zone lymphoma (MZL) and follicular lymphoma (FL).

The once-daily, oral, dual inhibitor of PI3K-delta and CK1-epsilon is for patients with previously treated MZL who have received at least one prior anti-CD20 based regimen and FL who have received at least two prior systemic therapies.

The MZL indication has a Prescription Drug User Fee Act (PDUFA) goal date of February 15, 2021, while the indication for FL has been accepted for standard review with a PDUFA goal date of June 15, 2021.

The NDA for umbralisib was based primarily on data from the umbralisib monotherapy MZL and FL cohorts of a Phase 2b trial. TG Therapeutics announced last year that each cohort met its primary endpoint of overall response rate (ORR), meeting the company’s target guidance of 40 to 50% ORR, as confirmed by an Independent Review Committee.

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“If approved, we believe umbralisib could become an important treatment option for patients with previously treated MZL and FL,” said Michael S. Weiss, executive chairman and CEO of TG Therapeutics in a 

. “This is a significant achievement in our path towards accomplishing our goal of developing novel treatments for patients with B-cell diseases.”

 the first-cell-based gene therapy to treat mantle cell lymphoma (MCL).

Brexucabtagene autoleucel (Tecartus, Kite, a Gilead company), a chimeric antigen receptor (CAR) T cell therapy, received Priority Review and Breakthrough Therapy Designation by FDA.

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The drug’s indication for marginal zone lymphoma (MZL) may not be approved until February 2021.
