Drexel University8d
Orphaned Again: Revisiting the Orphan Drug Act and Prioritizing Patient Access to Medicine
In 1983, Congress passed the Orphan Drug Act (“ODA”) as part of an effort to provide market incentives for pharmaceutical manufacturers to produce “orphan drugs,” drugs for diseases that ...
Fierce Pharma2d
Argenx's Vyvgart tops Trinity’s latest launch success analysis, beating out several Big Pharma debuts
Treatments for autoimmune diseases and those in orphan markets are on the up and up, as are drugmakers’ first launches, ...
Pharmaceutical Technology8d
Orphan drugs: How to avoid costly stock losses in Europe
With orphan drugs typically being high value, the potential risk of stock losses should be a top concern for biotechs looking ...
GlobalData on MSN3d
Lundbeck’s amlenetug gains orphan drug status in Japan for MSA
Lundbeck has gained orphan drug designation (ODD) from Japan’s Ministry of Health, Labor and Welfare (MHLW) for amlenetug to ...
Healio2d
FDA grants fast track designation to X-linked retinoschisis treatment
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release ...
The Manila Times22h
Cellectis Reports Financial Results for the Fourth Quarter and Full Year 2024 and Provides a Business Update
In 2025, Cellectis will continue to focus its efforts and expenses on advancing its core clinical trials BALLI-01 and NATHALI ...
GlobalData on MSN7d
FDA ODD given to Plus Therapeutics’ injectable radiotherapy
The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Plus Therapeutics’ injectable ...