News

EC grants orphan drug designation to Dyne Therapeutics’ DMD therapy
The company anticipates submitting a biologics licence application for accelerated approval in the US in early 2026.
AB Science announces that EMA granted orphan drug status to AB8939 for the treatment of acute myeloid leukemia
HAS GRANTED ORPHAN DRUG DESIGNATION IN THE EUROPEAN UNION FOR AB8939 IN THE TREATMENT OF ACUTE MYELOID LEUKEMIA Paris, April 23, 2025, 6.45pm CET AB Science ...
MPR1d
Dual EP2/EP4 Antagonist Designated Orphan Drug for Familial Adenomatous Polyposis
There are currently no FDA-approved treatments for FAP, a condition characterized by the proliferation of polyps in the colon and/or rectum.
NS Pharma gets FDA orphan drug status for autoimmune disease
NS Pharma, a unit of Nippon Shinyaku (OTCPK:NPNKF), said on Monday that the U.S. FDA has granted orphan drug designation to ...
Dyne Therapeutics receives EMA orphan drug designation for DYNE-251
Dyne Therapeutics (DYN) announced that the European Commission has granted orphan drug designation for DYNE-251 for the treatment of Duchenne ...
To speed up delivery, govt exempts orphan drugs from testing at ports
To ensure timely access to life-saving orphan drugs for patients, the government has exempted mandatory sampling and testing ...
NeuroNOS Granted FDA Orphan Drug Designation for Phelan-McDermid Syndrome, a Neurodevelopmental Disorder Linked to Autism
The FDA designation marks a significant milestone in the development of a novel treatment for a rare neurodevelopmental disorderPlan to initiate ...
MPR2d
Nitric Oxide-Regulating Therapy Gets Orphan Drug Status for Syndromic Autism
The Food and Drug Administration (FDA) has granted Orphan Drug designation to BA-102 for the treatment of Phelan-McDermid syndrome, a rare neurodevelopmental disorder linked to autism.
Tempest Receives Orphan Drug Designation from the FDA for TPST-1495 to Treat Patients with FAP
Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class1 targeted and ...
Using a pea-sized node in the brain to potentially treat drug addiction: Q&A
Major scientific discoveries can arise from simple decisions: say, by simply looking where virtually no one else has. Such ...
Dyne Therapeutics stock rises on orphan drug status
The orphan drug designation is particularly noteworthy as it can provide Dyne with a range of benefits, including reduced regulatory fees, assistance with clinical protocols, eligibility for research ...