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 ...

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 ...

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

Paris: Sanofi has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to Riliprubart ...

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 ...

ABBV-706 is an ADC targeting SEZ6 developed by AbbVie. It is linked to a topoisomerase 1 inhibitor (TOP1i) via a ...

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

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 ...

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 ...