FDA Updates for Week of Feb. 20, 2023

FDA approves weekly hemophilia A therapy.

The FDA has approved Altuviiio to treat patients with hemophilia A, a rare bleeding disorder. It occurs in about one in 5,000 male births annually, and more rarely in females. Previously referred to as efanesoctocog alfa, Altuviiio is a novel, recombinant factor VIII replacement therapy indicated for the prevention and on-demand treatment to control bleeding episodes in adults and children with hemophilia A. It is designed to extend protection from bleeds with once-weekly prophylactic dosing.

Sanofi officials expect Altuviiio to be available beginning in April 2023. They would not specify a list price until the product is commercially available but a spokesperson said Sanofi will price Altuviiio at parity to the annual cost of treating a prophylaxis patient on Eloctate, another recombinant factor VIII from Sanofi/Sobi to treat patients with hemophilia A. Sanofi provides Eloctate at $2.38 per IU, and company officials would not be more specific. Maryland’s Medicaid system indicated that in 2020, Elocate was one of the top 10 drugs in spending, with $951,166 spent for Eloctate 3,000 unit.

The spokesperson said Sanofi will provide support and resources to patients, including disease and medication education, electronic enrollment, financial assistance, insurance investigation paired with ePrescribing technology, and ongoing support to help address barriers throughout the treatment journey.

FDA approves extended-release Austedo.

The FDA has approved an extended-release version of Teva Pharmaceuticals’ Austedo (deutetrabenazine), a new once-daily formulation to treat adults with tardive dyskinesia (TD) and chorea associated with Huntington’s disease (HD).

Tardive dyskinesia is a chronic movement disorder that affects one in four people who take certain mental health treatments. It is characterized by uncontrollable, abnormal, and repetitive movements of the face, torso, and/or other body parts. Huntington’s disease is a fatal neurodegenerative disease. Chorea – involuntary, random and sudden, twisting and/or writhing movements – is one of the most striking physical manifestations of Huntington’s disease and occurs in about 90% of patients.

FDA grants accelerated approval to Filspari for rare kidney disease.

The FDA has granted accelerated approval to Travere Therapeutics’ Filspari (sparsentan) to reduce proteinuria, protein in the urine, in adults with primary IgAN. IgA nephropathy (IgAN), also called Berger's disease, is a rare progressive kidney disease characterized by the buildup of immunoglobulin A, a protein that helps the body fight infections, in the kidneys. The deposits of IgA cause a breakdown of the normal filtering mechanisms in the kidney, leading to blood in the urine (hematuria), protein in the urine, and a progressive loss of kidney function. It effects up to 150,000 people in the United States.

Filspari is a first-in-class, once-daily, oral medication designed to selectively target two critical pathways in the disease progression of IgAN (endothelin-1 and angiotensin II).

Filpari is expected to be available beginning the week of Feb. 27, 2023. Travere is offering Filspari at a wholesale acquisition cost of $9,900 for a 30-day supply. Travere will be providing a comprehensive patient support program with $0 copay for qualified patients.

Because of the risk of liver abnormalities associated with Filpari, it will be administrated through a Risk Evaluation and Mitigation Strategies program. Liver monitoring will be required monthly for the first year of treatment and then every three months. Additionally, monthly pregnancy monitoring will be required of all because of the risk to the embryo.

FDA approves first drug for geographic atrophy due to AMD.

The FDA has approved Apellis’ Syfovre (pegcetacoplan) to treat patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Geographic atrophy is an advanced form of age-related macular degeneration and a leading cause of blindness that impacts more than 5 million people worldwide, including 1 million people in the United States.

Syfovre is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system. It is designed to be injected into the back of the eye. Syfovre will be available by the beginning of March through specialty distributors and specialty pharmacies. It will have a list price of $2,190 per vial, and a spokesperson said the price reflects the Syfovre clinical profile and is in line with recently approved anti-VEGF therapies.

Apellis officials anticipate Medicare to be the dominant payer for patients with geographic atrophy because of the age of diagnosis of these patients. The company is helping patients with treatment access and support. ApellisAssist provides insurance support, financial assistance for eligible patients, disease education, and ongoing product support.

FDA to hold advisory committee meeting on Onpattro for heart failure indication.

The FDA has accepted Alnylam Pharmaceutical’s supplemental new drug application (sNDA) for Onpattro (patisiran), an RNAi therapeutic to treat the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. ATTR amyloidosis is a rare, rapidly progressive, debilitating disease caused by misfolded transthyretin proteins that accumulate as amyloid fibrils in multiple tissues including the nerves, heart, and gastrointestinal tract.

The FDA has set an action date of Oct. 8, 2023, under the Prescription Drug User Fee Act (PDUFA). The agency has not identified any review issues and said it would hold an advisory committee meeting to discuss the application.

Onpattro, already approved by the FDA to treat the polyneuropathy of hereditary ATTR amyloidosis in adults – which affects the peripheral nerves — netted $558 million in revenue in 2022. Its wholesale acquisition cost is $9,785 per vial.

FDA grants priority review for Pfizer’s RSV vaccine.

The FDA has accepted for priority review Pfizer’s biologics license application (BLA) for the syncytial virus (RSV) vaccine candidate PF-06928316 or RSVpreF to prevent lower respiratory tract illness caused by RSV in infants. This decision follows the FDA’s Breakthrough Therapy Designation for RSVpreF in March 2022. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of August 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 people and can potentially cause severe illness in young infants, older adults, and individuals with certain chronic medical conditions. Among children younger than five years old in the United States, RSV infections account for approximately 2.1 million outpatient visits and 58,000 hospitalizations each year. Worldwide, RSV results in the death of about 102,000 children annually. Among adults 65 years and older, RSV infections account for about 60,000 to 160,000 hospitalizations and between 6,000 and 10,000 deaths each year in the United States.

Pfizer’s RSVpreF is bivalent vaccine candidate is composed of equal amounts of recombinant RSV prefusion F from subgroups A and B. It is designed to be given to pregnant women to prevent lower respiratory tract illness caused by RSV in infants from birth up to six months of age

FDA grants priority review for therapy for life-threatening hereditary immune disease.

The FDA has accepted for priority review Regeneron’s biologics license application (BLA) for pozelimab to treat adults and children as young as 1 year of age with CHAPLE disease, an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system. There are currently no approved treatments for CHAPLE. Those living with CHAPLE are unable to regulate complement activity due to mutations in the CD55 gene, a protein regulating the body’s complement system. Without proper CD55 regulation, the complement system may attack normal cells, causing damage to blood and lymph vessels along the upper digestive tract and leading to the loss of proteins and to blood cells. There are fewer than 100 patients worldwide who are known to have CHAPLE.

Pozelimab is a fully human monoclonal antibody designed to block the activity of complement factor C5, a protein involved in complement system activation. The target action date for the FDA decision is Aug. 20, 2023.

FDA sets goal date for blurry vision therapy.

The FDA has accepted for review Orasis Pharmaceuticals’ new drug application (NDA) for CSF-1 to treat people with presbyopia, or age-related blurry vision. CSF-1 is a preservative-free formulation of low-dose pilocarpine administered as an eye drop. The agency has assigned a Prescription Drug User Fee Act (PDUFA) goal date of Oct. 22, 2023.

Presbyopia is the loss of ability to focus on near objects as a result of the natural aging process. It occurs mostly after the age of 40 when the crystalline lens of the eye gradually stiffens and loses flexibility. Almost 2 billion people globally and more than 120 million people in the United States live with presbyopia.