 Exelixis’ Cabometyx (cabozantinib) for the treatment of adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer that has progressed following treatment with a vascular endothelial growth factor receptor (VEGFR)-targeted therapy and who are resistant to radioactive iodine treatment.

This is more than two months ahead of the Prescription Drug User Fee Act (PDUFA) target action date of December 4, 2021. DTC is the most common type of thyroid cancer in the United States, and patients who are resistant to radioactive iodine treatment face a poor prognosis.

The approval is based on results from COSMIC-311, a phase 3 pivotal trial. At an interim analysis, Cabometyx reduce the risk of progression or death. At a follow-up analysis, the median progression-free survival was 11.0 months.

Serious adverse events seen in at least 5% of patients included hypertension, fatigue, diarrhea, and palmar-plantar erythrodysesthesia, which is a side effect that can cause redness, swelling, and pain in the hands and feet.

“Before today, patients with radioactive iodine-refractory differentiated thyroid cancer who have progressed following prior VEGFR-targeted therapy were facing aggressive disease and no standard treatment option,” Marcia S. Brose, M.D., Ph.D., chief, cancer center operation, Sidney Kimmel Cancer Center at Jefferson Torresdale Hospital, co-director, community based clinical trials, Sidney Kimmel Cancer Center at Thomas Jefferson University, and principal investigator of COSMIC-311, said in a statement.

In February 2021, the FDA granted Breakthrough Therapy Designation to Cabometyx for this indication.

Cabometyx is also approved for the treatment of patients with renal cell carcinoma (RCC), hepatocellular carcinoma who have been previously treated with Nexavar (sorafenib); and for patients with advanced RCC as a first-line treatment in combination with Opdivo (nivolumab).

The American Cancer Society estimates that 44,280 new cases of thyroid cancer will be diagnosed in the United States this year. Most (73%) of those cases will be in women. About 2,200 people will die from the disease.

Articles

The approval comes more than two months ahead of its PDUFA target date of December 4, 2021.


FDA Approves Cabometryx for Thyroid Cancer

Formulary Watch Drug Shortage Tracker — September 19, 2021

Denise Myshko is your editorial contact for the Formulary Watch. She joined Formulary Watch as editor is June 2021 from Oncology Live. Prior to that, she was managing editor of PharmaVOICE, a trade publication in the pharmaceutical industry. Email: dmyshko@mjhlifesciences.com

The FDA’s Vaccines and Related Biological Products Advisory Committee voted 16-2 against full approval for the use of Pfizer/BioNTech’s COVID-19 vaccine, Comirnaty, in people over the age of 16. But they did vote unanimously to provide an emergency use authorization of the vaccine as a booster after six months for those over the age of 65 and those at high risk of severe COVID-19.

Committee members recognized research that showed waning immunity from the COVID vaccines over time, but they indicated there was not enough data to support a third dose in the general population. They were concerned about the safety of the third dose of the vaccine, specifically heart inflammation, and they wanted to see data specifically on this area of safety.

Materials the FDA released before the meeting included 

 of real-world data in Israel that found a third dose of the vaccine boosted vaccine effectiveness to the level initially seen after the second dose. Investigators found that the rate of confirmed infection was lower in the booster group than in the nonbooster group by a factor of 11.3, and the rate of severe illness was 7.5 cases per 100,000 person-days.

The third dose of the Pfizer vaccine in Israel was approved on July 30, and investigators analyzed data through Aug. 31, 2021. This study was 

FDA advisory committee members were concerned about myocarditis in younger people given the third dose. Investigators in Israeli study found that out of about 2 million booster doses, a small number of people experienced serious adverse events.

In the United States, safety surveillance reports received by FDA and CDC identified serious risks for myocarditis and pericarditis following administration of the first two doses of the vaccine, with higher rates seen in men under the age of 40 with the highest rates among male adolescents 16 and 17 years of age, according to a 

. An FDA analysis of the Optum database estimated an excess risk approaching 200 cases per million vaccinated males 16 and 17 years of age, but information was not available on long-term outcomes related to myocarditis.

When polled, all committee members agreed when polled to extending the EUA booster to those at risk for occupational exposure, including health care workers.

 the EUA for a booster of the Pfizer vaccine to those at least 12 years of age who have undergone an organ transplant, or who have conditions that lead to a similar level of level of compromised immune system. This authorization is separate and distinct from the booster dose reviewed by the committee.


The vaccines advisory committee voted late on Friday, September 17, 2021, to provide emergency authorization of Pfizer’s COVID-19 vaccine as a booster for those over the age of 65, as well as for those at high risk of developing severe disease. 


FDA Advisory Committee Supports COVID-19 Boosters for Some People

 applications to the FDA and the EMA for Rinvoq (upadacitinib) for the treatment of adults with moderate-to-severe ulcerative colitis. The applications are supported by data from two phase 3 induction studies and one maintenance study.

In these studies, more patients treated with Rinvoq achieved the primary endpoint of clinical remission and all secondary end points compared with placebo with 45 mg once daily at week eight for the induction studies, and with 15 mg and 30 mg once daily at 52 weeks for the maintenance study.

 of Rinvoq 15 mg or 30 mg were consistent with the safety profile observed in the phase 3 induction studies in ulcerative colitis, as well as in previous studies across indications. No new safety risks were identified.

“Many people continue to struggle with symptoms associated with their ulcerative colitis, such as fatigue, bowel urgency, bloody diarrhea and abdominal pain,” Tom Hudson, M.D., senior vice president of research and development, chief scientific officer, AbbVie, said in a statement. "Upadacitinib has the potential to be an important new treatment option for people with ulcerative colitis, who want to address these challenging and disruptive symptoms. We look forward to working with regulatory authorities and hope to bring upadacitinib to people with ulcerative colitis as quickly as possible."

Ulcerative colitis is a chronic inflammatory bowel disease of the large intestine. In 2015, an estimated 1.3% of U.S. adults (3 million) reported being diagnosed with inflammatory bowel disease, either Crohn’s disease or ulcerative colitis, 

Rinvoq is a JAK inhibitor that is being studied in several immune-mediated inflammatory diseases. Rinvoq 15 mg is approved in the United States for adults with moderate to severe rheumatoid arthritis. It is approved in the EU for adults with moderate to severe active rheumatoid arthritis, adults with active psoriatic arthritis, adults with active ankylosing spondylitis (AS), and adults and adolescents 12 years and older with moderate to severe atopic dermatitis (AD). Rinvoq 30 mg is approved in the EU for adults with moderate to severe atopic dermatitis under age 65.

Related: FDA’s New Warning Includes Xeljanz and other JAK Inhibitors

Rinvoq achieved primary and secondary end points compared with placebo.


AbbVie Submits Rinvoq to FDA and EMA for Ulcerative Colitis

 all remaining lots of Chantix (varenicline) 0.5 mg and 1 mg tablets because of the presence of a nitrosamine, which may be associated with an increased risk of cancer.

Chantix is a treatment to help patients quit smoking and is intended for short term use. The products were distributed nationwide to wholesalers and distributors in the United States, U.S. Virgin Islands, and Puerto Rico from May 2019 to September 2021.

The NDC, lot numbers, and expiration date affected can be found 

 made over the last few months when 16 lots of Chantix had been recalled.

Pfizer officials say they are making this move as precaution since other options are now available. Last month, the FDA 

 the first generic version of Chantix. Developed by Par Pharmaceutical, which is part of Endo International, the generic varenicline tablet was approved August 11, 2021. The generic will come in doses of 0.5 mg and 1 mg.

Additionally, In July after Pfizer’s initial recalls, Apotex Corp., in coordination with the FDA, 

 temporary importation of Apo-Varenicline (varenicline tartrate) 0.5 mg and 1 mg tablets into the U.S. market. Apo-Varenicline is the varenicline tartrate product approved by Health Canada as a generic equivalent to Champix tablets manufactured by Pfizer Canada. Champix is the brand name of Chantix in Canada.


To date, Pfizer has not received reports of adverse events related to this recall, but is making the move as a precaution. 



Pfizer Recalls All Lots of Smoking Cessation Drug Chantix

 Eli Lilly’s emergency use authorization (EUA) for bamlanivimab 700 mg and etesevimab 1400 mg administered together to include post-exposure prophylaxis.

The neutralizing antibodies can now be used together to treat high-risk individuals 12 years of age and older who have not been fully vaccinated against COVID-19 or are not expected to mount an adequate immune response to complete vaccination, and have been exposed to someone infected with SARS-CoV-2.

 for the U.S. government to purchase 388,000 doses of etesevimab to complement doses of bamlanivimab previously purchased by the U.S. government, with about 200,000 doses expected to ship third quarter of 2021 and the remaining to be shipped in fourth quarter. This transaction is expected to generate about $330 million in revenue in the second half of 2021, according to a Lilly statement.

"The recent increase in COVID-19 cases has caused a substantial rise in the utilization of monoclonal antibody drugs, particularly in areas of the country with low vaccination rates," Daniel Skovronsky, M.D., Ph.D., Lilly's chief scientific and medical officer, and president of Lilly Research Laboratories, said in a statement. "Lilly developed bamlanivimab and etesevimab for administration together, in anticipation of variants such as the highly contagious Delta variant, which currently accounts for more than 98 percent of all identified COVID-19 cases in the United States."

The expanded authorization is based on data from BLAZE-2, a study conducted in partnership with the National Institute of Allergy and Infectious Diseases and the COVID-19 Prevention Network that enrolled residents and staff at long-term care facilities across the United States. In this placebo-controlled phase 3 study, bamlanivimab reduced the risk of contracting symptomatic COVID-19 by up to 80% in nursing home residents and up to 57% among residents and staff of long-term care facilities.

Bamlanivimab and etesevimab together are authorized for the treatment of mild-to-moderate COVID-19 in adults and adolescents (12 years of age) with positive results of direct SARS-CoV-2 viral testing. But the combination is not authorized for use in patient who are hospitalized or who need oxygen therapy.

Bamlanivimab is a recombinant, neutralizing human IgG1 monoclonal antibody directed against the spike protein of SARS-CoV-2. It was designed to block viral attachment and entry into human cells. Etesevimab is a recombinant fully human monoclonal neutralizing antibody that specifically binds to the SARS-CoV-2 surface spike protein receptor and can block the binding of the virus to the ACE2 host cell surface receptor.

The combination of bamlanivimab/etesevimab can now be given to both treat and prevent COVID-19 infection after exposure.

FDA Expands EUA for Lilly’s COVID-19 Monoclonal Antibody

Regeneron has chosen Amber Specialty Pharmacy and Hy-Vee Pharmacy Solutions for 

 administration of Regeneron’s REGEN-COV (casirivimab and imdevimab) monoclonal antibody to treat and prevent COVID-19. The organizations have 21 locations throughout the United States and Puerto Rico. All locations are licensed to administer the REGEN-COV antibody treatment. A list of locations can be found 

REGEN-COV is was approved under an Emergency Use Authorization to both treat non-hospitalized patients and prevent infection from COVID-19. In July, Regeneron had received an 

 for the post-exposure prophylaxis in people exposed to COVID-19. This was supported by phase 3 data showing 81% reduced risk of symptomatic infections in close contacts of those who have COVID-19.

 that the U.S. Department of Health and Human Services and the Department of Defense will purchase 1.4 million additional doses of REGEN-COV. The government will continue to provide REGEN-COV at no cost to patients. Under the new agreement, Regeneron will supply the additional 1,200 mg doses of REGEN-COV to the U.S. government by January 31, 2022, at a cost of $2,100 per dose. This new agreement follows two earlier agreements with the U.S. government announced in 

Regeneron expects to begin delivering the additional REGEN-COV doses to the U.S. government in September, with the vast majority delivered in the fourth quarter of 2021.

"While vaccination remains the first line of defense to decrease the burden of COVID-19, REGEN-COV is a key tool that reduces the risk of hospitalization or death by 70% in high-risk individuals when given early in the course of the infection. Recently there has been greater demand for REGEN-COV, and we will provide additional doses to the U.S. government as quickly as possible,” Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron, said in a statement.

The company continues development of the antibody treatment for the prevention of symptomatic infection in asymptomatic household contacts (both 

) of SARS-CoV-2 infected individuals; treatment of 

 patients already infected with SARS-CoV-2, and treatment of certain patients 

 due to COVID-19 infection, including the UK 

REGEN-COV is a cocktail of two monoclonal antibodies that was designed specifically to block infectivity of SARS-CoV-2 using Regeneron’s proprietary 

Regeneron also announced an expanded contract with the U.S. government for the purchase of additional doses of REGEN-COV.

Amber Specialty, Hy-Vee to Dispense Regeneron Monoclonal Antibody for COVID-19

 Takeda Oncology’s Exkivity (mobocertinib) to treat adult patients with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

Exkivity, which was granted priority review and received breakthrough therapy designation, fast track designation, and orphan drug designation from the FDA, is the first approved oral therapy to target EGFR exon 20 insertion mutations.

This indication is approved under accelerated approval based on overall response rate and duration of response. The FDA approval is based on results from the platinum-pretreated population in the phase 1/2 trial of Exkivity in patients who had received prior platinum-based therapy. Results were presented at the 

2021 American Society of Clinical Oncology (ASCO) Annual Meeting

 In May. In this trial, the therapy demonstrated an overall response rate of 28% as well as a median duration of response of 17.5 months, a median overall survival of 24 months.

The most common adverse reactions were diarrhea, rash, nausea, vomiting, decreased appetite, fatigue, dry skin, and musculoskeletal pain.

“EGFR exon20 insertion NSCLC is an underserved cancer that we have been unable to target effectively with traditional EGFR tyrosine kinase inhibitors,” Pasi A. Jänne, M.D., Ph.D., director of the Lowe Center for Thoracic Oncology at Dana Farber Cancer Institute, said in a statement. “The approval of EXKIVITY (mobocertinib) marks another important step forward that provides physicians and their patients with a new targeted oral therapy specifically designed for this patient population that has shown clinically meaningful and sustained responses.”

 Thermo Fisher Scientific’s Oncomine Dx Target Test as a companion diagnostic to identify patients who are candidates for Exkivity. The test evaluates 23 genes associated with NSCLC. The FDA first approved the test as a companion diagnostic in 2017. In the United States, it is now approved for five targeted therapies for non-small cell lung cancer and one targeted therapy for cholangiocarcinoma.

Non-small cell lung cancer is the most common form of lung cancer, 

 about 85%. The global cancer burden is expected to be 28.4 million cases in 2040, a 47% rise from 2020, according to one 

.Patients with EGFR exon 20 insertions make up about 1% to 2% of patients with non-small cell lung cancer.

Another therapy for exon 20 insertion mutations was approved earlier this year for this indication. In May 2021, the FDA 

 Rybrevant (amivantamab-vmjw), a bispecific antibody directed against EGFR and MET receptors. Developed by Janssen, the drug is used to treat adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutation who have progressed with chemotherapy. Rybrevant, which is administered intravenously, had previously received the FDA’s priority review and breakthrough therapy designation.

 Guardant Health’s Guardant360 CDx liquid biopsy as a companion diagnostic.


Takeda Oncology’s Exkivity treats non-small lung cancer in patients with EGFR exon 20 insertion mutations. This is the second approval for this mutation and the first oral therapy.



FDA Approves Exkivity, a Novel Oral Therapy for Lung Cancer

The FDA has accepted Axsome Therapeutics’ new drug application (NDA) for meloxicam-rizatriptan (AXS-07) for the treatment of acute migraine, bringing the novel investigational medication one step closer to full FDA approval.

Meloxicam-rizatriptan is a novel, oral, rapidly absorbed therapy for migraine, Axsome said in a 

. It is designed to provide rapid, enhanced and consistent relief of migraine, with reduced symptom recurrence, according to the company.


The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2022, for the NDA. 

“The FDA’s acceptance of the NDA for AXS-07 is an important milestone for Axsome as it brings us closer to potentially making this multi-mechanistic treatment available to migraine patients in need,” Herriot Tabuteau, M.D., CEO of Axsome, said in a statement.

The NDA is supported by results from two phase 3 randomized, double-blind, controlled trials that demonstrated elimination of migraine pain compared with placebo and active controls, the pharma maker said.

Migraines are common condition, with 15.3% of Americans aged 18 years or older reporting a migraine or severe headache in the previous three months, 

Meloxicam is a new molecular entity enabled by Axsome’s MoSEIC (Molecular Solubility Enhanced Inclusion Complex) technology, which results in rapid absorption while maintaining a long half-life. Meloxicam is a COX-2 preferential non-steroidal anti-inflammatory drug and rizatriptan is a 5-HT1B/1D agonist.

Meanwhile, OptumRx listed another investigational migraine medication, AbbVie’s ategoepant, as one of 

 for the third quarter of 2021. Ategoepant, expected to garner FDA approval in the third quarter, offers an additional oral treatment option for preventing migraine attacks.

The FDA has set a Prescription Drug User Fee Act target action date of April 30, 2022.

Investigational Oral Migraine Treatment Closer to Full FDA Approval

 BeiGene’s Brukinsa (zanubrutinib) for the treatment of adult patients with relapsed or refractory marginal zone lymphoma who have received at least one anti-CD20-based regimen.

Brukinsa is a small molecule inhibitor of Bruton’s tyrosine kinase, which plays a role in B-cell receptor signaling, a driver in the development of marginal zone lymphoma. In 2016, there were 

 7,460 newly diagnosed patients with marginal zone lymphoma in the United States.

The approval is based on efficacy results from two single-arm clinical trials, with overall response rate as the primary end point. In the phase 2 MAGNOLIA trial, 66 patients were evaluated. Based on assessment using CT scan, the overall response rate was 56%; based on assessment prioritizing PET-CT scan, the overall response rate was 67%. In a phase 1/2 trial, 20 patients were evaluated, and based on assessment using CT scan, the overall response rate was 80%.

The most common adverse reactions in a pooled safety population of 847 patients were decreased neutrophil count, upper respiratory tract infection, decreased platelet count, hemorrhage, decreased lymphocyte count, rash, and musculoskeletal pain.

“We are optimistic that Brukinsa will bring clinically meaningful benefit to patients with relapsed or refractory marginal zone lymphoma,” Stephen Opat, MBBS, director of clinical hematology at Monash Health, head of department of hematology at Monash University, and lead principal investigator of the MAGNOLIA trial, said in a statement.

The recommended dose is either 160 mg twice daily or 320 mg once daily, taken orally with or without food.

Brukinsa was approved by the FDA in November 2019 for the treatment of mantle cell lymphoma in adult patients who have received at least one prior therapy and in August 2021 for adult patients with Waldenström’s macroglobulinemia.

The agency has granted accelerated approval to Brukinsa as a second-line treatment for patients with marginal zone lymphoma, a group of slow-growing non-Hodgkin lymphomas.

Brukinsa Snags Third FDA Approval for Lymphoma

 for filing Marinus Pharmaceuticals’ new drug application (NDA) for the use of ganaxolone in the treatment of seizures associated with CDKL5 deficiency disorder, a rare, genetic epilepsy. The NDA was granted Priority Review designation and the FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of March 20, 2022.

The application is supported by data from the Marigold study, a phase 3 trial in 101 patients. Patients treated with ganaxolone showed a 30.7% median reduction in 28-day major motor seizure frequency, compared with a 6.9% reduction for those receiving placebo. Patients in the open-label extension study treated with ganaxolone for at least 12 months experienced a median 49.6% reduction in major motor seizure frequency.

Ganaxolone received orphan drug designation in 

 and a Rare Pediatric Disease designation for CDKL5 deficiency disorder in 

“The FDA’s acceptance of our NDA submission is an important step toward potentially bringing the first approved therapy specifically for treatment of seizures associated with CDD—a devastating disorder with high unmet medical need—to families and healthcare providers,” Scott Braunstein, M.D., chief executive officer of Marinus Pharmaceuticals, said in a statement. “We believe that ganaxolone has the potential to provide meaningful clinical benefit for patients and we look forward to working closely with the FDA during the review process.”

 that is caused by a mutation of the cyclin‑dependent kinase‑like 5 (CDKL5) gene, located on the X chromosome. CDKL5 deficiency disorder is characterized by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment. Currently, there are no therapies approved specifically for CDD. The incidence 1 in 40,000 to 60,000 newborns. About 90% of those diagnosed with CDKL5 deficiency disorder are girls.

The FDA assigned a PDUFA action date of March 20, 2022, for ganaxolone.


FDA has Accepted Application for Rare Epilepsy

 to the FDA seeking approval for Skyrizi (risankizumab-rzaa), an interleukin-23 (IL-23) inhibitor, for the treatment of patients 16 years and older with moderate-to-severe Crohn’s disease. Approval is being sought for both the 600 mg intravenous induction and 360 mg subcutaneous maintenance therapy.

The submission is supported by safety and efficacy data from three phase 3 studies – ADVANCE, MOTIVATE and FORTIFY. In these trials, Skyrizi demonstrated improvements in clinical remission and endoscopic response as both induction and maintenance therapy. The overall safety findings in these studies were consistent with previous studies.

In the FORTIFY study, a significantly greater proportion of participants achieved the co-primary end points of endoscopic response and clinical remission with Skyrizi subcutaneous maintenance therapy at one year (52 weeks) for both assessed doses (360 mg or 180 mg), compared with those who were randomized to the withdrawal arm and received placebo. 

In the ADVANCE and MOTIVATE induction studies, a significantly greater proportion of patients with Crohn's disease treated with either dose of Skyrizi IV induction therapy (600 mg or 1200 mg) met the co-primary end points of clinical remission and endoscopic response at week 12 compared with placebo.

“While there have been advancements in care, many people with Crohn's disease do not achieve lasting remission,” Tom Hudson, senior vice president of research and development, chief scientific officer, AbbVie, said in a statement.

In April 2019, the FDA approved Skyrizi for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. Phase 3 trials of Skyrizi in psoriatic arthritis, Crohn's disease, and ulcerative colitis are ongoing.

 is a chronic disorder that primarily affects the digestive system. This condition involves an abnormal immune response that causes excess inflammation. It most often affects the intestinal walls, particularly in the lower part of the small intestine and portions of the large intestine. Crohn’s disease is most common in western Europe and North America, where it has a prevalence of 100 to 300 per 100,000 people. More than half a million Americans are currently affected by this disorder.

In trials, Skyrizi demonstrated improvements in clinical remission and endoscopic response as both induction and maintenance therapy

AbbVie Submits Skyrisi to FDA for Crohn’s Disease

 priority review of the Biologics License Application (BLA) for the combination of relatlimab and Opdivo (nivolumab), administered as a single infusion, for the treatment of adult and adolescents with unresectable or metastatic melanoma. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 19, 2022.

The BLA submission was based on the efficacy and safety results of the phase 2/3 RELATIVITY-047 trial, which demonstrated that the combination therapy achieved progression-free survival benefit of 10.1 months compared with 4.6 months for Opdivo alone.

“We believe that the relatlimab and nivolumab fixed-dose combination has the potential to improve outcomes for patients with metastatic or unresectable melanoma,” Jonathan Cheng, senior vice president and head of oncology development, Bristol Myers Squibb, said in a statement.

Relatlimab is the first LAG-3-blocking antibody to demonstrate a clinical benefit for patients with phase 3 data. Primary results from the RELATIVITY-047 trial were 

 during the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2021. Adverse events leading to discontinuation of treatment in 14.6% of patient receiving the combination therapy versus 6.7% for those receiving Opdivo alone.

Lymphocyte-activation gene 3 (LAG-3) functions to control T-cell response, activation, and growth.

Melanoma is the fifth most common cancer, 

 to the National Institutes of Health. The NIH estimates that in 2021 more than 7,000 people will die from melanoma.

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 19, 2022.

FDA Accepts BMS Application for Opdivo/Relatimab Combination for Advanced Melanoma

 Biogen and Samsung Bioepis’ Byooviz (ranibizumab-nuna) as the first biosimilar to Genentech’s Lucentis (ranibizumab injection) for the treatment of several eye diseases and conditions, including neovascular (wet) age-related macular degeneration, a leading cause of vision loss and blindness for Americans over the age of 65. Byooviz is also approved to treat macular edema following retinal vein occlusion (blockage of veins in the retina) and myopic choroidal neovascularization, a vision-threatening complication of myopia (nearsightedness).

Byooviz is an anti-vascular endothelial growth factor (VEGF) therapy that prevents vision loss in patients with retinal vascular disorders, which can cause irreversible blindness or visual impairments in adults in the United States.

The FDA approval of Byooviz was based on a totality of evidence including analytical, nonclinical data, and clinical data. In a phase 3 study, the efficacy, safety, pharmacokinetics, and immunogenicity of Byooviz was compared with Lucentis in patients with wet AMD.

“Today’s approval provides another treatment option for millions of people whose vision is impaired and is another step forward in our commitment to provide access to safe, effective and high-quality biological products,” Sarah Yim, M.D., director of the Office of Therapeutic Biologics and Biosimilars in the FDA’s Center for Drug Evaluation and Research, said in a statement.

There are two types of AMD—dry and wet. While dry AMD is more common, wet AMD leads to vision loss at a faster rate. Both macular edema and myopic choroidal neovascularization can also lead to visual impairment. About 11 million people 

 with AMD in the United States alone, with a global prevalence of 170 million.

Byooviz was approved in the European Union (EU) on August 18, 2021, and the United Kingdom on August 31, 2021.

BYOOVIZ is Samsung Bioepis’ fifth biosimilar approved in the United States, following the approval of Renflexis (infliximab-abda), a biosimilar of Remicade approved in April 2017 to treat several autoimmune diseases including Crohn’s and rheumatoid, Ontruzant (trastuzumab-dttb), a biosimilar of Herceptin approved in January 2019 to treat HER2-positive breast and gastric cancer, Eticovo (etanercept-ykro), a biosimilar of Enbrel approved in April 2019 to treat arthritis and plaque psoriasis, and Hadlima (adalimumab-bwwd), a biosimilar of Humira approved in July 2019 to treat arthritis and psoriasis.

Biogen/Samsung Bioepis’ Byooviz is a biosimilar of Genentech’s Lucentis. 

FDA Approves First Biosimilar to Treat Macular Degeneration 

The Centers for Medicare & Medicaid Services (CMS) 

 a permanent J-code (J2406) for Melinta Therapeutics’ Kimyrsa (oritavancin), a lipoglycopeptide antibiotic, effective October 1, 2021. This code allows the product to be reimbursed when dispensed in the outpatient setting. Kimyrsa is approved to treat acute bacterial skin and skin structure infections in a single, one-hour, 1,200 mg infusion. The FDA approved the therapy on March 12, 2021, for the treatment of adult patients with Gram-positive infections, including methicillin-resistant Staphylococcus aureus (MRSA).

At the same time, CMS has granted transitional pass-through status for Kimyrsa in the hospital outpatient department setting. Pass-through status is intended to protect Medicare beneficiaries’ access to innovative drugs and ensures that Kimyrsa will be reimbursed at average sales price for three years. Drugs with pass-through status are not subject to the payment reduction applicable to drugs purchased through the 340B program.

“We intentionally designed Kimyrsa to provide patients with more flexibility and accessibility in acute bacterial skin and skin structure infection treatment. These achievements are one more significant step toward our vision that all patients who need our therapies will be able to receive them,” Christine Ann Miller, Melinta’s president and chief executive officer, said in a statement.

Bacterial skin and skin structure infections affect about 14 million patients in the United States each year.

Effective October 1, 2021, CMS will reimburse for the infusion product when dispensed in the outpatient setting.

FDA has Accepted Application for Rare Epilepsy

AbbVie Submits Skyrisi to FDA for Crohn’s Disease

FDA Accepts BMS Application for Opdivo/Relatimab Combination for Advanced Melanoma

FDA Approves First Biosimilar to Treat Macular Degeneration

CMS Assigns J-Code for the Antibiotic Kimyrsa