Advances in Rare Disease Therapies: Orphan Drugs, Trials, and More

The FDA granted Orphan Drug Designation to Veverimer for anti-GBM kidney disease and to Vasceptor, a non-opioid gel for sickle cell pain. Capricor’s Duchenne therapy Deramiocel advances with Becker designation and a clean FDA pre-license inspection. Chiesi partnered with Key2Brain on enzyme therapies for rare CNS disorders. Angitia’s AGA2115 showed early promise for brittle bone disease. Circle Pharma’s CID-078 targets small cell lung cancer. Novartis’ Fabhalta improved outcomes in PNH trials. Meanwhile, the Senate dropped the Orphan Cures Act, prompting biopharma backlash and patient group support.

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