The FDA rejected Novo Nordisk’s biologics license application for icodec, citing questions about the manufacturing process for this once weekly insulin as well as its use by type 1 diabetes patients. But there are several high-profile FDA approvals in our recap of recent regulatory news.

By Frank VinluanNovo Nordisk’s bid to bring diabetes patients a less burdensome dosing regimen has encountered a setback. The FDA turned down the company’s application for icodec, a slow-acting insulin the company designed for once-weekly dosing as an alternative to daily insulin injections.
According to Novo Nordisk, the FDA’s complete response letter raised questions about icodec’s manufacturing process as well as the use of the product in patients with type 1 diabetes. In May, an FDA advisory committee concluded that the available data were not sufficient to show that icodec’s benefits outweigh its risks in type 1 diabetes. Some committee members expressed concerns about icodec’s higher rates of hypoglycemia, or low blood sugar, compared with daily insulin injections. The committee did not discuss use of icodec in type 2 diabetes. Novo Nordisk said it is reviewing the FDA letter but does not expect to be able to address the agency’s requests this year.
Rival Eli Lilly is developing a once-weekly insulin called efsitora alfa. In May, the company reported clinical data showing this insulin was comparable to daily insulin injections in type 2 diabetes patients, meeting the main goal of that Phase 2 study.
Icodec has approvals in both type 1 and type 2 diabetes in Europe, Canada, Australia, Japan, and Switzerland, where the product is marketed under the brand name Awiqli. In China, this weekly insulin is approved only for type 2 diabetes.
Here’s a recap of other recent regulatory news:
—Arcutis Biotherapeutics’ topical drug Zoryve is now approved as a treatment for mild-to-moderate atopic dermatitis. The product’s latest approval covers the treatment of adults as well as children age 6 and older. Zoryve works by blocking PDE-4, an enzyme involved in itching and inflammation in the skin. Arcutis first won approval for the product in 2022 for treating plaque psoriasis. Late last year, a topical foam formulation of the drug was approved for seborrheic dermatitis.
—Kisunla, an anti-amyloid drug developed by Eli Lilly, received FDA approval for treating patients in the early stages of Alzheimer’s disease. The drug will compete with Eisai’s Leqembi, an amyloid plaque-busting therapy that received its FDA approval last year.
—Johnon & Johnson’s blockbuster immunology drug Stelara is getting more biosimilar competition. Pyzchiva, developed by Samsung Bioepis, is now FDA approved for all indications covered by the J&J product, including plaque psoriasis, psoriatic arthritis, and Crohn’s disease. Pyzchiva will be commercialized in the U.S. by Sandoz. Approval of Pyzchiva follows the 2023 regulatory nod for Wezlana, a Stelara biosimilar developed by Amgen.
—The FDA approved Verona Pharma drug Ohtuvayre as a maintenance treatment for chronic obstructive pulmonary disease (COPD). The once-daily pill is a single small molecule designed to block two enzyme targets associated with the chronic respiratory disease. Ohtuvayre is the first novel COPD medication to pass FDA muster in more than a decade.
—The FDA turned down Rocket Pharmaceuticals’ application for Kresladi, a gene therapy developed for severe leukocyte adhesion deficiency-I (LAD-I). The Cranbury, New Jersey-based company said the FDA has requested limited additional chemistry manufacturing and controls information to complete its review. LAD-I is an inherited disorder that leads to severe, life-threatening bacterial and fungal infections. Currently, the only curative treatment is a bone marrow transplant.
—Chronic inflammatory demyelinating polyneuropathy (CIDP), a disease that affects nerves and leads to muscle weakness, now has its first FDA-approved treatment. Argenx drug Vyvgart Hytrulo won the regulatory nod for treating the rare autoimmune condition. The injectable drug was initially approved for treating a different nerve and muscle disease called generalized myasthenia gravis.
—The FDA approved a Sarepta Therapeutics gene therapy that failed the main goal of its Phase 3 clinical trial, achieving only secondary goals. FDA reviewers said the results on secondary goals were unclear about whether the patient benefit was due to the therapy. Top FDA biologics official Peter Marks overruled agency staff, concluding that the results show clinical benefit.
—The FDA is taking comments regarding a potential change in its switching study requirements for biosimilars. Biosimilars cannot currently be substituted for a brand name biologic unless the follow-on medication has interchangeability status. That status requires data from studies showing there’s no difference in switching between the products. The FDA proposes doing away with the switching study requirement.
—Bristol Myers Squibb drug Augtyro now has an expanded label allowing its use for treating any type of advanced solid tumor carrying a particular genetic signature, an NTRK gene fusion. The drug won its initial approval last November as a treatment for non-small cell lung cancer characterized by ROS1 gene fusions. BMS added Augtyro to its pipeline via the $4.1 billion acquisition of the drug’s developer, Turning Point Therapeutics.
—Amgen drug blinatumomab, brand name Blincyto, won a new FDA approval as a treatment for Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia, a type of blood and bone marrow cancer. The FDA decision covers the treatment of this cancer that’s positive for CD-19, the protein that Blincyto targets. Blincyto is a T cell engager, a type of therapy that works by binding to a T cell and a cancer cell simultaneously. The Amgen drug won its first FDA approval in 2014 as a treatment for precursor B-cell acute lymphoblastic leukemia.
—The FDA approved Yimmugo, a Grifols treatment for primary immunodeficiencies. The intravenously infused immunoglobulin therapy was developed by a Grifols subsidiary, Biotest. Grifols develops therapies based on proteins sourced from human plasma. The company plans a second half 2024 U.S. launch for Yimmugo, which has been commercially available in Europe since 2022.
—Tris Pharma received FDA approval for its generic version of deflazacort (brand name Emflaza), a Duchenne muscular dystrophy drug marketed by PTC Therapeutics. PTC offers its drug in tablet and liquid formulations. The Tris drug will be available in liquid form. It will be commercialized by Cranbury Pharmaceuticals, a subsidiary that the company has formed to market its more than 20 generic medicines. Tris recently formed Tris Digital Health, a subsidiary that will focus on developing digital products for neurological indications. Tris Pharma will remain focused on attention deficit hyperactivity disorder, pain, neurological diseases, and addiction.
—Speaking of PTC Therapeutics, the New Jersey drugmaker encountered another setback in Europe for a different Duchenne therapy, Translarna. The European Commission’s Committee on Medicinal Products for Human Health (CHMP) issued another negative opinion on the renewal of marketing authorization for the therapy.
Translarna’s conditional approval requires PTC to seek annual renewals until the company produces clinical data to support standard marketing authorization. The drug’s Phase 3 failure led the CHMP to recommend against renewal last year and again early this year. In May, the commission directed the CHMP to reconsider, taking into account the “totality of evidence,” including patient registries and real-world evidence. Following the latest CHMP action, PTC said it will request a re-examination of the opinion.
Photo: Getty Images, Sarah Silbiger