FDA Updates for Week of Feb. 27, 2023


The FDA has approved Kevzara for an inflammatory rheumatic disease and Skyclarys, the first therapy for rare neuromuscular disease. The agency set an action date for Opdivo for melanoma indication. An advisory committee has supported Pfizer’s RSV vaccine, and Janssen has submitted an NDA for combo tablet for metastatic prostate cancer.

FDA approves Kevzara for inflammatory rheumatic disease.

The FDA has approved Kevzara (sarilumab) to treat adult patients with polymyalgia rheumatica (PMR), an inflammatory rheumatic disease. It is indicated for patients who have had an inadequate response to corticosteroids or who cannot tolerate corticosteroid taper.

PMR often initially presents with pain and stiffness around the neck, shoulder, and hip area and symptoms include fatigue, low-grade fever, and weight loss. Patients often experience flares during tapering of, or relapse after discontinuation of corticosteroid treatment. PMR generally affects people who are 50 years and older.

Developed by Regeneron and Sanofi, Kevzara is currently approved in multiple countries to treat adults with moderately to severely active rheumatoid arthritis who have not responded to or tolerated previous therapy. Kevzara binds specifically to the IL-6 receptor and has been shown to inhibit IL-6-mediated signaling. IL-6 is an immune system protein produced in increased quantities in patients with rheumatoid arthritis.

Kevzara has an average retail price of $5,554.56 for one carton of two syringes, according to GoodRx. Patients with commercial insurance can apply for $0 copay, up to an annual maximum of $15,000, through the KevzaraConnect program. The company also offer a free, one-time 30-day supply, as well as a patient assistance program for uninsured and underinsured.

FDA approves first therapy for rare neuromuscular disease.

The FDA has approved Skyclarys (omaveloxolone) to treat patients with Friedreich’s ataxia in adults and adolescents aged 16 years and older. With this approval, the FDA granted a rare pediatric disease priority review voucher.

Friedreich’s ataxia is an ultra-rare, inherited neurodegenerative disorder that is typically diagnosed during adolescence. Patients with Friedreich’s ataxia experience progressive loss of coordination, muscle weakness, and fatigue, which commonly progresses to motor incapacitation and wheelchair reliance and eventually death. Friedreich’s ataxia affects about 5,000 diagnosed patients in the United States.

Developed by Reata Pharmaceuticals, Skyclarys oral, once-daily activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling.

Skyclarys will be available in the second quarter of 2023 with a wholesale acquisition cost of $370,000. The company is offering Skyclarys through a patient support program, ReataReach, which will provide insurance and access support and commercial copay support. The company anticipates Skyclarys will be placed on a specialty tier, Dawn Bir, chief commercial officer, said in a conference. They anticipate that 60% of patients will have commercial insurance Reata will offer a $0 copay card, but regardless of coverage, no patient will face more than a nominal copay, Bir said.

FDA advisory committee supports Pfizer’s RSV vaccine.

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted that available data is adequate to support the safety and effectiveness of Pfizer’s respiratory syncytial virus (RSV) bivalent vaccine candidate PF-06928316 or RSVpreF. The committee voted 7 to 4 on safety and 7 to 4 on effectiveness. The vaccine is currently under FDA review to prevent acute respiratory disease and lower respiratory tract disease caused by RSV in adults 60 years of age and older. An FDA decision is expected in May 2023.

RSV is a contagious virus and a common cause of respiratory illness. The virus can affect the lungs and breathing passages of an infected individual and can potentially cause severe illness in young infants, older adults, and those with certain chronic medical conditions. In the United States alone, among older adults, RSV infections account for about 60,000 to 160,000 hospitalizations and 6,000 to 13,000 deaths each year.

Pfizer’s bivalent vaccine candidate is composed of two preF proteins selected to optimize protection against RSV A and B strains. The vaccine builds on discoveries including those made at the National Institutes of Health (NIH), which detailed the crystal structure of prefusion F, a key form of the viral fusion protein that RSV uses to enter human cell

FDA sets action date for Opdivo for melanoma indication.

The FDA has accepted the supplemental biologics license application (sBLA) for Opdivo (nivolumab) as monotherapy to treat patients with stage IIB or IIC melanoma after surgery. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) date of Oct. 13, 2023.

Melanoma is a form of skin cancer characterized by the uncontrolled growth of pigment-producing cells located in the skin. In the United States, about 97,610 new diagnoses of melanoma and about 7,990 related deaths are estimated for 2023. Melanomas can be mostly treatable when caught in very early stages; however, survival rates can decrease as the disease progresses.

Developed by Bristol Myers Squibb, Opdivo is PD-1 immune checkpoint inhibitor that is designed to harness the body’s own immune system to help restore anti-tumor immune response. It is already indicated for patients 12 years of age or older with melanoma with involvement of lymph nodes or metastatic disease. Opdivo-based therapies are also approved for other tumors, including non-small cell lung cancer (NSCLC), bladder cancer and esophageal/gastroesophageal junction cancer.

Janssen submits NDA for combo tablet for metastatic prostate cancer.

Janssen Pharmaceutical has submitted a new drug application (NDA) to the FDA seeking approval of niraparib/abiraterone as a dual-action tablet to treat patients with BRCA-positive metastatic castration-resistant prostate cancer (mCRPC). If approved, this will be the first dual-action tablet formulation for patients with mCRPC with BRCA mutations, which are a type of homologous recombination repair (HRR) gene alteration.

Metastatic castration-resistant prostate cancer is a cancer that has spread beyond the prostate gland and has progressed despite medical or surgical treatment to lower testosterone. Patients with mCRPC and HRR gene alterations are more likely to have aggressive disease, poor outcomes and a shorter survival time. The combination of niraparib, a highly selective poly (ADP-ribose) polymerase (PARP) inhibitor, and abiraterone acetate, a prodrug of abiraterone, a CYP17 inhibitor, plus prednisone, targets two oncogenic drivers in patients with mCRPC: the androgen receptor axis and HRR gene alterations. Through the dual action tablet formulation, this therapeutic option may help improve compliance and reduce pill burden.

The NDA is supported by data from the MAGNITUDE study, a phase 3 study evaluating the safety and efficacy of the combinational therapy as a first-line treatment in patients with mCRPC, with or without alterations in HRR associated genes.

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