Orphan Drug, FDA and Korro

Korro Bio gets Orphan Drug Designation from FDA for its genetic disorder treatment
Korro Bio (NASDAQ:KRRO) said on Friday that it received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat alpha-1 antitrypsin deficiency. Stock is up about 10% premarket.
KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
KRRO-110 is the first RNA editing development candidate from Korro’s proprietary OPERA TM platform and is currently being evaluated in the Phase 1/2a REWRITE clinical study for AATD. Dosing of the first two single ascending dose cohorts in healthy adult volunteers has been completed, and an interim readout is expected in the second half of 2025.
Korro Bio's KRRO-110 Gets FDA Orphan Drug Designation
Korro Bio disclosed that the Food and Drug Administration granted orphan drug designation to its potential genetic disorder drug KRRO-110. Shares rose 17.6% to $29 in premarket trading. The stock is down about 60% over the last year.
Lundbeck’ amlenetug receives Japanese orphan drug designation for treatment of multiple system atrophy
Lundbeck’ amlenetug receives Japanese orphan drug designation for treatment of multiple system atrophy: Denmark Tuesday, March 11, 2025, 11:00 Hrs [IST] H. Lundbeck A/S (Lundbec
DiagnaMed Granted FDA Orphan Drug Designation for Molecular Hydrogen to Treat Amyotrophic Lateral Sclerosis
"Receiving orphan drug designation from the FDA is a significant milestone for DiagnaMed and validates the promise of molecular hydrogen as a potential treatment for ALS. We are excited to partner with Revive Therapeutics to advance this program and meet the urgent needs of the ALS community."
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 impact ...
GlobalData on MSN3d
Lundbeck’s amlenetug gains orphan drug status in Japan for MSA
Amlenetug is tailored to attach to all major extracellular α-synuclein forms. The human monoclonal antibody aims to prevent ...
NeuroSense’s ALS drug PrimeC shows success in phase 2b, eyes final trial
PrimeC has been given an ‘Orphan Drug’ status by the FDA (Food and Drug Administration) and EMA (European Medicines Agency).
PharmiWeb8d
Global Orphan Drug Market Poised for Significant Growth Amid Rising Demand and Regulatory Support
The global orphan drug market size was USD 154.20 Billion in 2022 and is expected to register a rapid revenue CAGR of 12.3 % ...
Targeted Oncology7d
FDA Grants Rhenium Obisbemeda Orphan Drug Status for LM in Lung Cancer
The FDA granted orphan drug status to rhenium-186 obisbemeda for treating leptomeningeal metastases in lung cancer.
Medical Device Network8d
Realising the opportunities for growth in the European orphan drug market
The European orphan drug market is growing and is already comparable in size and scope to the US market. The European Union ...
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, ...