Pharmaceutical Industry Update: FDA Repurposing, Major Acquisitions, and Psychedelics Revival

In a flurry of activity, the pharmaceutical industry has seen significant developments across multiple fronts, from regulatory shifts to high-profile acquisitions and emerging therapeutic areas.

FDA Targets Autism with Drug Repurposing Initiative

The U.S. Food and Drug Administration (FDA) has announced plans to repurpose GSK's Wellcovorin (leucovorin) for cerebral folate deficiency, a condition linked to "developmental delays with autistic features." This move follows a recent press conference where President Donald Trump, alongside Health Secretary Robert F. Kennedy Jr., suggested a potential connection between Tylenol use during pregnancy and rising autism rates.

Pfizer's Strategic Acquisition and Industry M&A Trends

Pfizer has made a bold move to reinvigorate its portfolio by acquiring obesity treatment startup Metsera for $4.9 billion. This acquisition comes after Pfizer faced setbacks with three discontinued assets over the past two years. The deal aligns with a broader industry trend of "bite-sized" acquisitions, exemplified by Roche's recent $3.5 billion agreement with 89bio for a metabolic dysfunction-associated steatohepatitis asset, and Novartis's $5.7 billion collaboration with Monte Rosa Therapeutics.

Psychedelics: The Next Frontier in Pharmaceutical M&A

The psychedelics sector is emerging as a hotbed for mergers and acquisitions, following AbbVie's $1.2 billion purchase of Gilgamesh Pharmaceuticals' lead depression asset. Industry experts speculate that major players like Eli Lilly, Bristol Myers Squibb, and Merck may be next to enter this burgeoning field.

Regulatory and Rare Disease Developments

The CDC's newly restructured vaccine advisory committee held its inaugural meeting, discussing schedules for COVID-19, MMRV, and hepatitis B vaccines. However, industry observers noted concerns about the committee's expertise and experience.

In the rare disease space, Stealth BioTherapeutics secured approval for elamipretide (now Forzinity) to treat Barth syndrome, a disorder that falls under the FDA's new Rare Disease Evidence Principles framework for ultra-rare diseases affecting fewer than 1,000 people in the U.S.