FDA Updates for the Week of Jan. 3, 2023

FDA approves Alzheimer’s medication lecanemab.

The FDA has issued an accelerated approval for Biogen/Eisai’s second Alzheimer’s disease (AD) medication, lecanemab. Now with the name brand name Leqembi, it is a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody approved to treat patients with mild cognitive impairment or mild dementia stage of disease. The approval was based on a surrogate marker, the ability to clear amyloid beta plaque in the brains of patients with Alzheimer’s disease.

Leqembi will have a wholesale acquisition cost of $26,500 a year — below the estimated $37,600 annual quantified societal value determined by Eisai executives — but still higher than the cost-effectiveness range of between $8,500 and $20,600 that the Institute for Clinical and Economic Review has suggested in a draft review. ICER analysts remain uncertain about using amyloid removal as a surrogate marker for clinical benefit in Alzheimer’s disease.

FDA approves new therapy for urea cycle disorders.

The FDA has approved Olpruva (sodium phenylbutyrate) to treat certain patients living with urea cycle disorders (UCDs), a genetic condition that causes ammonia to build up in the blood. This ca can lead to brain damage and neurocognitive impairments. Developed by Acer Therapeutics and Relief Therapeutics, Olpruva is a new formulation of sodium phenylbutyrate packaged as a premeasured, single dose packet. It is indicated for patients who have deficiencies of the specific enzymes that are responsible for removing ammonia from the blood, including carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinic acid synthetase.

The approval was made under the 505(b)(2) section of the Food, Drug and Cosmetic Act, which allows for companies to use third-party data for approval. In its application, Acer cited preclinical and clinical safety and efficacy data from the reference listed drug, Buphenyl powder, which is approved as adjunctive therapy in the chronic management of patients with urea cycle disorders.

Wegovy approved for obesity in teens.

Even though the FDA has approved a new indication for Novo Nordisk’s diabetes medication Wegovy (semaglutide) injection — the first and only anti-obesity drug for teens — it may take a while for all Wegovy users to obtain supply.

Wegovy, approved in June 2021 for chronic weight management in adults with obesity or overweight with at least one weight-related condition (such as high blood pressure, type 2 diabetes, or high cholesterol), is now also indicated for use in patients 12 years old and older, Novo Nordisk said in a news release.

Wegovy, however, is one of the diabetes and weight loss medications that have been in short supply. Novo Nordisk’s Ozempic (semaglutide) and Lilly’s Trulicity (dulaglutide) and Mounjaro (tirzepatide) were all listed on FDA's Drug Shortages database in December 2022.

FDA grants priority review for glofitamab for blood cancer.

The FDA has accepted Genentech’s biologics license application (BLA) and granted priority review for glofitamab to treat adult patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) after two or more lines of systemic therapy. Large B-cell lymphoma is a fast-growing non-Hodgkin’s lymphoma (NHL) and is one of the most prevalent types of blood cancer among adults in the United States.

Glofitamab is a T-cell engaging bispecific antibody that targets CD3 on the surface of T cells and CD20 on the surface of B cells. It was engineered to have one region that binds to CD3, a protein on T cells, and two regions that bind to CD20, a protein on B cell. This dual-targeting brings the T cell in close proximity to the B cell, activating the release of cancer cell-killing proteins from the T cell.

If approved, it would be the first off-the-shelf T-cell engaging bispecific antibody to treat people with an aggressive lymphoma who have previously received multiple courses of treatment. The FDA is expected to make a decision on approval by July 1, 2023.

FDA accepts second UCB application for generalized myasthenia gravis.

The FDA has accepted for priority review UCB’s biologic license application (BLA) rozanolixizumab, a monoclonal antibody to treat adults with generalized myasthenia gravis (gMG). Generalized myasthenia gravis is chronic and unpredictable autoimmune disease that causes weakness in the skeletal muscles, including those in the arms and legs and those involved in breathing. The condition worsens with activity.

Rozanolixizumab is a subcutaneous monoclonal antibody targeting the neonatal Fc receptor (FcRn). If approved, it would be indicated for patients who are positive for the anti-acetycholine receptor (AChR) or the anti-muscle-specific tyrosine kinase (MuSK) antibody. Rozanolixizumab was also accepted for review by the European Medicines Agency. Decisions from both agencies are expected in the second quarter of 2023.

This is the second therapy from UCB to be accepted for FDA review to treat patients with generalized myasthenia gravis. Zilucoplan, subcutaneous, self-administered peptide inhibitor of complement component 5, is a targeted therapy that inhibits key components in the underlying pathophysiology of generalized myasthenia gravis. The anticipated PDUFA date is in the fourth quarter of 2023.

FDA accepts BLA for RSV prevention in infants.

The FDA has accepted a biologics license application (BLA) for nirsevimab to prevent respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and children up to 24 months of age. Nirsevimab is being developed and commercialized by AstraZeneca in collaboration with Sanofi. It is a single-dose, long-acting antibody designed to protect infants entering or during their first RSV season and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season.

The Prescription Drug User Fee Act date is in the third quarter of 2023. If approved at that time, nirsevimab will be available in the United States for the 2023/2024 RSV season.

Celltrion submits BLA for new formulation of infliximab.

Celltrion USA has submitted to the FDA a biologics license application (BLA) for lead product candidate, CT-P13 SC, which is a subcutaneous formulation of infliximab to be used a maintenance therapy for patients with inflammatory bowel disease (IBD). If approved, this would be the first subcutaneous formulation of infliximab.

A subcutaneous formulation has the potential to enhance treatment options for the use of the infliximab drug by providing high consistency in drug exposure and a convenient method of administration. “CT-P13 SC releases the burden of having to travel to treatment for IV infusions, reducing treatment-related travel costs for patients and caregivers,” Jaeik Shim, chief operating officer of Celltrion USA, said in a press release.

Celltrion was the first company to develop a biosimilar of Janssen’s Remicade (infliximab). The FDA approved Inflectra (infliximab-dyyb) in April 2016 to treat patients with several conditions, including Crohn’s, ulcerative colitis, rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis and plaque psoriasis. Both Remicade and Inflectra are administered by intravenous infusion.

Orasis submits NDA for blurry vision treatment.

Orasis Pharmaceuticals has submitted a new drug application (NDA) to the FDA for investigational 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.

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.

If approved, this would be the second therapy to treat patients with presbyopia. The first FDA- approved therapy is Vuity, which was developed by Allergan, an AbbVie company. Vuity is a daily, prescription eye drop that works lasts up to 6 hours. Vuity uses a technology allows the therapy to rapidly adjust to the physiologic pH of the tear film.