Retail chatter around Crinetics Pharmaceuticals picked up late Sunday after the company released long-term data for its oral ...

President Donald Trump’s One Big Beautiful Bill, signed into law last week, reintroduces broader exemptions for orphan drugs ...

Retail chatter around Klotho Neurosciences picked up late Thursday after the company said that the FDA had granted Orphan Drug Designation to its ALS gene therapy candidate, KLTO-202. The designation ...

The FDA has granted orphan status to Arcus Biosciences’ CD73 inhibitor quemliclustat for metastatic pancreatic cancer, as the ...

ADRX-0405 is uniquely being tested during its 1a/b clinical trials. ADRX-0405 is labeled as a STEAP1 ADC (primarily ...

US FDA grants orphan drug designation to Klotho Neurosciences’ KLTO-202 to treat amyotrophic lateral sclerosis: New York Saturday, July 12, 2025, 15:00 Hrs [IST] Klotho Neurosci ...

The GOP tax bill repeals the Inflation Reduction Act’s price controls for drugs for rare diseases.

Arcus Biosciences' cancer drug candidate, quemliclustat, earns FDA orphan drug status, ensuring incentives and exclusivity.

Hospitals could be on the hook for significantly higher outpatient drug prices. Because of the cuts to Medicaid, hospitals in the 340B program will see fewer Medicaid patients and may risk losing ...

Of the 43 blockbuster brand name drugs that raked in over $1 billion in annual profits, 18 fell under the orphan drug category. Even smaller biopharmaceutical enterprises are getting in on the action.

There are around 7,000 rare diseases for which there currently exists no authorised or satisfactory method of treatment, highlighting a vast area of unmet need, and yet the clinical development of ...

Half of the recent acquisitions among drug makers and biotechs involved an orphan drug, according to Moody’s Investor Service, which believes the trend will continue.