Breaking with its long-held, oft-cited mantra that observational studies are great for generating hypotheses but not for approvals, the U.S. FDA is initiating the approval of leucovorin calcium tablets for patients with cerebral folate deficiency (CFD), a neurological condition that affects folate transfer into the brain. Developmental delays with autistic features, seizures, and problems with movement and coordination have been observed in patients with this deficiency. The agency said the new indication for the drug, which has gone generic, is based on a systematic analysis of literature published between 2009-2024, including case reports with patient-level information, and mechanistic data. While the indication would be for CFD, President Donald Trump called leucovorin “a potential drug for some autism symptoms” at a media briefing late yesterday.

Biotech leaders say macroeconomics and US policy shifts are making M&A hard

The unsettling impact that Trump administration’s health care and tariffs initiatives are having on M&A activity and financing is highlighted in a survey of CEOs and investors, who say uncertainty related to the U.S. policy environment is the top challenge they face when it comes to executing deals. Of 397 respondents from around the world asked to name their three main concerns, 57% cited the U.S. policy environment. Meanwhile, U.S. drug pricing and reimbursement was an issue for 39%, while 32% of respondents to the survey, carried out by the investment bank Lazard, cited FDA regulatory uncertainty. Compared to the previous year, concerns about the FDA have risen steeply, with only 12% of respondents having said regulatory uncertainty was a restraint on dealmaking in the 2024 survey.

Another Catalent delay: Scholar Rock gets CRL for apitegromab

A failed July inspection of manufacturer Catalent Indiana LLC has delayed another U.S. FDA approval, the latest being that of Scholar Rock Inc.’s selective anti-latent myostatin antibody, apitegromab, which was expected to become the first therapy to enhance skeletal muscle in patients with spinal muscular atrophy. Despite the complete response letter (CRL) received by the company, analysts believe an eventual approval is likely, with the CRL noting no other issues. Cantor analyst Eric Schmidt said the news was disappointing, as the company was “expecting outright approval as recently as yesterday evening,” on the Sept. 22 PDUFA date. Scholar Rock’s shares were trading at $32.33, down 25 cents, at midday Sept. 23.

CRL halts Otsuka’s drive for a PTSD treatment

The first new U.S. FDA-approved therapeutic option for PTSD in more than 20 years will have to wait. Rexulti (brexpiprazole), combined with sertraline, from one of Japan’s biggest pharmas, Otsuka Pharmaceutical Co. Ltd., is on hold with a complete response letter from the agency. The company said the letter states that the FDA has completed its review but cannot approve the application in the current form and that the sNDA does not provide substantial evidence of effectiveness to support the approval. In July, in a 10-1 vote, the FDA’s Psychopharmacologic Drugs Advisory Committee said the treatment did not show enough efficacy.

Australia bets on mRNA as Moderna builds regional hub

While U.S. policymakers are scaling back funding on mRNA vaccine research, Australia is accelerating investment in the technology. At the center of this effort is Moderna Inc., which is establishing an mRNA research and manufacturing footprint in the country and banking on decades of local expertise to bring new therapies to the clinic and serve as a regional hub for Asia Pacific. Australia’s first large-scale mRNA manufacturing facility is expected to deliver economic and health dividends – with modeling estimated to boost GDP by AU$220 million (US$145 million) a year, support nearly 1,000 ongoing jobs, and deliver pandemic preparedness benefits valued at up to AU$4.8 billion over 30 years, according to a new report by Oxford Economics.

Celltrion to buy Lilly’s cGMP plant for $330M, invest up to $1B

Celltrion Inc. announced plans to acquire from Eli Lilly and Co. a Branchburg, N.J.-based biologics cGMP facility for $330 million up front, with plans to invest up to a total of ₩1.4 trillion (US$1 billion) in plant acquisition and expansion. Incheon, South Korea-based Celltrion noted that the plant acquisition is part of a comprehensive tariff response strategy outlined by Celltrion group Chairman Seo Jung-jin in May.

More stem cell, gene therapies progressing in clinic for ALS

Amyotrophic lateral sclerosis (ALS), formerly known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disorder with no known cure. While three therapies have gained U.S. FDA approvals to date, including Rilutek (riluzole), Radicava/Radicava ORS (edaravone) and tofersen (BIIB-067), the lack of a disease-modifying drug has spurred the continual search for novel therapies. “Conventional ALS drug discovery has been based on a pathology-center approach, or a single-target approach [for symptomatic patients],” Oh Ki-wook, professor of neurology at Hanyang University’s College of Medicine, said at the Rare Genetic Disease Symposium in Busan, South Korea, noting that “alternative strategies are now emerging in South Korea, including stem cell therapy … and gene therapy.” 

Also in the news

Alvotech, Amgen, Aptose, Astrazeneca, Ausper, Ausperbio, Avalo, Avenzo, Betaglue, C4X, Chiesi, Ensoma, Evecxia, Genfit, Gilead, Hanmi, Incyte, Johnson & Johnson, Maplight, Mavrix, Merck, Myricx, Qualigen, Regeneron, Revive, Sanofi, Satellos, Servier, Stealth, Teva