Browsing Tag
rare disease
21 posts
GSK to sell linerixibat rights to Alfasigma for up to $690m as rare liver disease drug nears FDA approval
GSK and Alfasigma agree worldwide licence for linerixibat, a rare liver disease itch drug nearing FDA approval. Deal worth up to $690 million. Read more.
CSL (ASX:CSL) breaks ground on $1.5bn Kankakee plasma plant expansion, betting patented Horizon 2 technology can widen its immunoglobulin lead
CSL (ASX:CSL) breaks ground on a $1.5B Kankakee plasma plant expansion using patented Horizon 2 yield tech. Read the full strategic analysis.
Pfizer-backed Priovant wins FDA priority review for brepocitinib in dermatomyositis
FDA grants priority review to Priovant's brepocitinib NDA for dermatomyositis with a Q3 2026 PDUFA date. Read what this means for Roivant, Pfizer, and the autoimmune market.
Can UPLIZNA transform gMG treatment? Amgen’s FDA win sets new benchmark for antibody-positive patients
Discover how Amgen’s UPLIZNA is redefining generalized myasthenia gravis treatment after FDA approval. Find out what this means for patients and investors.
Can Ascendis Pharma’s TransCon CNP still win FDA approval after the review extension?
FDA delays decision on Ascendis Pharma’s dwarfism drug TransCon CNP to February 2026 due to protocol revisions. See how this affects approval odds and launch.
MeiraGTx signs $475m ophthalmology gene therapy collaboration with Eli Lilly
MeiraGTx Holdings plc signed a USD 475 million gene therapy deal with Eli Lilly and Company to develop AAV-AIPL1 and apply riboswitch tech in ophthalmology.
Rhythm Pharmaceuticals’ IMCIVREE hits regulatory speed bump as FDA extends decision timeline into 2026
Find out how Rhythm Pharmaceuticals’ IMCIVREE FDA review delay could shape the rare obesity treatment market in 2026.
Palatin Technologies to raise $15.8m in public offering; NYSE relisting expected Nov 12
Palatin Technologies regains NYSE American compliance and secures $15.8M for its oral MC4R obesity drug. Find out how the relisting and IND impact its biotech trajectory.
Breakthrough hope: Benitec Biopharma’s BB-301 delivers promising results in rare muscular dystrophy trial
Find out how Benitec Biopharma’s BB-301 gene therapy is showing early success in restoring swallowing function for OPMD patients.
Belite Bio wins UK MHRA nod to file conditional marketing application for tinlarebant in Stargardt disease
Find out how Belite Bio gained UK MHRA backing to file a conditional marketing application for tinlarebant, advancing the first potential therapy for Stargardt disease.