Bright Skies, Big Challenges: Orphan Drugs and Rare Diseases

FDA approved an at-home induction option for risankizumab-rzaa in adults with moderate-to-severe Crohn's disease, based on the phase 3 AFFIRM trial.

Treasury proposes adding biotechnology to its Outbound Investment Security Program, a pharma funding roundup covers Gilead’s $1.275 billion acquisition of an oral IL-23R inhibitor from PepStar and Zymeworks’ licensing deal with Roche, and Incubate’s John Stanford on why MFN is an offer pharma can’t refuse.

Dave Maraldo, Ph.D., president of manufacturing at Merck, explains how flexible capacity, earlier input from manufacturing in R&D, and digital tools are helping the organization keep up with a faster, more varied portfolio.

The Treasury Department is proposing rules to add biotechnology to its Outbound Investment Security Program under the COINS Act.

Gilead agrees to acquire an oral IL-23R inhibitor from PepStar, while Zymeworks licenses its DLL3-targeting T cell engager ZW209 to Roche.

Incubate’s John Stanford on why MFN is voluntary in name only — and why the moonshot is the first casualty of drug pricing pressure.

FDA grants Ultomiris accelerated approval for primary immunoglobulin A nephropathy, a pharma funding roundup covers Alcon’s $550 million acquisition of Neurotech and AI Proteins’ collaboration with Astellas, and Incubate’s John Stanford on which therapeutic areas MFN deals might hit hardest.

Alcon is acquiring Neurotech Pharmaceuticals to add the only FDA-approved MacTel cell therapy to its retina portfolio, while AI Proteins has enters a collaboration with Astellas to design novel miniprotein therapeutics.