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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.

Markets Insider · 20h

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.

MarketWatch · 19h

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.

Yahoo Finance · 10d

The US FDA Grants Both Orphan Drug and Rare Pediatric Disease Designations to NEU-001 of Neurenati for the Treatment of Hirschsprung Disease

Neurenati Therapeutics Inc., a biotech company focused on rare pediatric diseases, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to NEU-001,

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, ...

GlobalData on MSN3d

Lundbeck’s amlenetug gains orphan drug status in Japan for MSA

Lundbeck has gained orphan drug designation (ODD) from Japan’s Ministry of Health, Labor and Welfare (MHLW) for amlenetug to ...

Healio2d

FDA grants fast track designation to X-linked retinoschisis treatment

The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release ...

Pharmaceutical Technology8d

Orphan drugs: How to avoid costly stock losses in Europe

With orphan drugs typically being high value, the potential risk of stock losses should be a top concern for biotechs looking ...

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 ...

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