A week after catching Moderna Inc. and its investors off guard with a refuse-to-file letter, the U.S. FDA has reversed course on the company’s BLA submission seeking approval of seasonal influenza vaccine mRNA-1010, now agreeing to review the application and setting an assigned PDUFA date of Aug. 5, 2026.
Though it’s largely viewed by analysts as a simple delay rather than a setback, Disc Medicine Inc.’s unexpected complete response letter (CRL) for bitopertin in the rare genetic disorder erythropoietic protoporphyria (EPP) raises more questions regarding consistency and stability at the U.S. FDA.
As its GLP-1 rival goes after compounders, Eli Lilly and Co. started the week with news beyond its diabetes and obesity franchise. The company, which disclosed a new agreement with Innovent Biologics Ltd., followed up a short time later with plans to acquire Orna Therapeutics Inc., marking its latest foray in the in vivo therapy space.
On the heels of an NDA submission for brepocitinib in dermatomyositis, Priovant Therapeutics Inc. disclosed data showing the dual TYK2/JAK1 inhibitor surpassed expectations in a phase II study in cutaneous sarcoidosis, another chronic inflammatory skin condition with limited treatment options, setting the stage for a phase III study set to start this year.
Reauthorization of the rare pediatric disease priority review voucher (PRV) was included in the spending package signed into law Feb. 3 by U.S. President Donald Trump, removing at least one aspect of uncertainty facing rare disease companies, many of which have relied on the PRV’s transferrable option as a lucrative source of funding.
It’s not often that disclosure of a complete response letter (CRL) causes a company’s share price to rise, but that was case for Aquestive Therapeutics Inc., which saw its stock (NASDAQ:AQST) rise 39% to close Feb. 2 at $4.10 on news the U.S. FDA has declined – for now – to approve the NDA for Anaphylm (dibutepinephrine), delaying the commercial entry of what could be the first oral alternative to Epipen.
Heeding the recommendation of an independent data monitoring committee, Calcimedica Inc. said it is discontinuing the phase II study testing calcium release-activated calcium channel inhibitor Auxora in patients with acute kidney injury with associated acute hypoxemic respiratory failure.
After a roller coaster of a year for Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics Inc. looks to focus on the efficacy narrative in 2026, starting with newly unveiled three-year data showing durable efficacy across all key motor function assessments for treated DMD patients vs. external controls.
Citing the “totality of data,” Sanofi SA plans to file global regulatory submissions this year for amlitelimab in atopic dermatitis, despite mixed results from two additional phase III trials testing the OX40-ligand (OX40L) inhibitor the pharma firm has been positioning as a potential successor to its Regeneron Pharmaceuticals Inc.-partnered blockbuster, Dupixent (dupilumab).
With rumors regarding a couple of potential mega-mergers making the rounds, the week of the annual J.P. Morgan Healthcare Conference kicked off with the official disclosure of some billion-dollar collaborations, leading with Abbvie Inc.’s exclusive licensing deal with Remegen Co. Ltd. for PD-1/VEGF-targeted bispecific antibody RC-148.
