Kiniksa stock rocketed 20% after first-quarter results on April 29. The biotech stock then broke out of a cup base with a buy ...

The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from ...

A provision protecting expensive medications, used to treat multiple rare diseases, from the Medicare drug price negotiations ...

The Orphan Cures Act has been added back into the Senate version of President Donald Trump’s “big, beautiful bill” after being ...

7 years of market exclusivity in the U.S. upon FDA approval ...

Paris: Sanofi has received orphan drug designation from the Ministry of Health, Labour and Welfare (MHLW) in Japan for ...

Sanofi (ENXTPA:SAN) recently received orphan drug designation from Japan's Ministry of Health for riliprubart, aimed at treating chronic inflammatory demyelinating polyneuropathy. This underscores the ...

Congenital adrenal hyperplasia (CAH) is a rare autosomal recessive disease1 with an estimated global incidence of approximately 1 in 14,000–18,000 live births2A phase II trial has recently been ...

Japanese MHLW grants orphan drug designation to Sanofi’s riliprubart for chronic inflammatory demyelinating polyneuropathy: Paris Tuesday, July 1, 2025, 09:00 Hrs [IST] The Mini ...

Riliprubart granted orphan drug designation in Japan for chronic inflammatory demyelinating polyneuropathy. Two phase 3 studies are currently underway testing riliprubart in peopl ...

Hemispherian’s GLIX1 to treat malignant glioma receives US FDA orphan drug designation: Oslo, Norway Thursday, July 3, 2025, 15:00 Hrs [IST] Hemispherian AS, an Oslo-based pharm ...

Expansion of the Medicare drug price negotiation rare disease drug carve-out would unnecessarily limit the number of drugs ...