EMA grants orphan drug status to Sanofi’s Efdoralprin Alfa for rare lung disease

Efdoralprin alfa, a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, showed superior efficacy to standard plasma-derived therapy in adults with AATD

SandboxAQ, MapLight team up to target novel CNS therapy with AI-driven drug discovery

SandboxAQ will receive an upfront payment and could earn up to $200 million in milestone payments tied to preclinical, development, regulatory, and commercial achievements

Gilead’s new HIV therapy shows promising Phase 3 results

The study found BIC/LEN to be statistically non-inferior to BIKTARVY, Gilead’s current standard-of-care therapy

Merck’s KEYTRUDA–Padcev combo delivers survival gains in Phase 3 bladder cancer trial

Vanda Pharmaceuticals seeks FDA nod for breakthrough psoriasis drug

Imsidolimab, an IgG4 IL-36 receptor antagonist, works by inhibiting IL-36 receptor signaling, directly addressing the deficiency in the IL-36RA regulator common in GPP patients

Anil Koul joins Mankind Pharma as Group Chief Scientific Officer

More than five drug candidates from his team progressed to human trials including drugs like Rilematovir (Phase IIb), and Bedaquiline

Merck wins key EU backing to expand use of PAH Drug WINREVAIR

WINREVAIR is currently the first and only activin signalling inhibitor approved for PAH across all 27 EU member states

Suven Life Sciences achieved 100% patient enrolment in Phase-2b clinical trial of Ropanicant for MDO

The Phase-2b study is a randomized, double-blind, placebo-controlled trial that will enroll approximately 195 patients across 35 sites in the USA

Neurizon’s ALS drug regimen cleared for launch in Healey platform trial

The Healey ALS Platform Trial is a multicenter, double-blind, placebo-controlled

Formation Bio snaps up global rights to next-gen CNS TYK2 inhibitor from Lynk Pharma

LNK01006 is designed to deliver potent, selective inhibition of TYK2-mediated cytokine signaling with central nervous system exposure