Novo Nordisk recently faced a setback as the FDA rejected their application for icodec, a diabetes treatment designed for once-weekly dosing instead of daily injections. The FDA’s concerns centered on the manufacturing process and the drug’s application for type 1 diabetes, with specific worries about heightened risks of hypoglycemia. Despite this hitch, icodec continues to be approved for use in European, Canadian, Australian, Japanese, and Swiss markets, and under the name Awiqli, for both type 1 and type 2 diabetes, with an exception in China where it’s only approved for type 2 diabetes.
In related developments, Eli Lilly, a competitor, is also advancing with its once-weekly insulin product, efsitora alfa, showing promising results in a Phase 2 study for type 2 diabetes, indicating equivalency to daily insulin injections.
Apart from insulin-related therapies, several other drugs and treatments across various categories have seen regulatory movements:
– Arcutis Biotherapeutics obtained approval for Zoryve, initially for plaque psoriasis and later for seborrheic dermatitis. Newly, it has been approved for mild-to-moderate atopic dermatitis in adults and children aged six and above.
– Eli Lilly’s Kisunla has been approved for early-stage Alzheimer’s disease, offering a new treatment option alongside Eisai’s earlier approved amyloid plaque-targeting therapy, Leqembi.
– Pyzchiva, a biosimilar to Johnson & Johnson’s immunology drug, Stelara, covering various conditions like plaque psoriasis and Crohn’s disease, was brought to market through a collaboration between Samsung Bioepis and Sandoz.
Innovative treatments for chronic diseases include the FDA’s approval of Verona Pharma’s Ohtuvayre, a novel chronic obstructive pulmonary disease (COPD) treatment, marking a significant development as the first novel COPD medication approved in over a decade.
Rocket Pharmaceuticals, however, faced a denial from the FDA for Kresladi, a gene therapy for leukocyte adhesion deficiency-I, due to the need for more manufacturing and control information.
The treatment spectrum widened for rare conditions with the approval of Vyvgart Hytrulo by Argenx for chronic inflammatory demyelinating polyneuropathy, following its prior approval for a different nerve and muscle disease.
Among biologic treatments, the FDA granted approval to a gene therapy by Sarepta Therapeutics for primary immunodeficiencies, despite the therapy not meeting its primary goals in Phase 3 clinical trials. Interestingly, top FDA biologics official Peter Marks overruled concerns, focusing on discerned clinical benefits.
The regulatory environment may also see some procedural easing as the FDA considers eliminating the requirement for switching studies in biosimilars, potentially simplifying market access for such treatments.
Treatment approvals extended in cancer therapies as well, with Bristol Myers Squibb’s Augtyro being greenlit for treating advanced solid tumors with a specific genetic marker, NTRK gene fusion, after its initial approval for a type of lung cancer. Amgen’s blinatumomab also expanded its approvals in leukemia treatments.
On the hormonal treatment front, Yimmugo, by Grifols, got a nod for treating primary immunodeficiencies and is planned for a U.S. launch in the latter half of 2024 after being available in Europe since 2022.
In generic medications, Tris Pharma gained approval for a liquid form of deflazacort, a Duchenne muscular dystrophy treatment, under the brand name Emflaza from PTC Therapeutics, with plans for market release by a newly formed subsidiary.
Conversely, PTC Therapeutics faced challenges in Europe with Translarna, a treatment for Duchenne muscular dystrophy, after failing to secure a renewal of its marketing authorization due to inadequate clinical data, though the company plans to request a re-examination based on comprehensive evidence.
These developments highlight a dynamic regulatory landscape where novel therapies are emerging alongside significant innovations in biosimilars and generics, pushing forward the boundaries of treatment across various medical conditions.
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