ASHBURN, Va. — Quoin Pharmaceuticals Ltd., a specialty pharmaceutical company focused on rare and orphan diseases, today announced that it has received U.S. Food and Drug Administration (FDA) clearance to implement a number of protocol amendments to its two ongoing clinical trials for QRX003, which is being developed as a...
treatment News
ASHBURN, Va. — Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) , a specialty pharmaceutical company focused on rare and orphan diseases, today announced clearance from the U.S. Food and Drug Administration (FDA) to recruit teen subjects aged 14 years and older into its two ongoing clinical trials for QRX003, which is being...
ASHBURN, Va. — Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX), a specialty pharmaceutical company exclusively focused on the development and commercialization of innovative therapies that treat rare and orphan diseases, today announced it has filed U.S. and International patent applications for a novel combination product as a potential treatment of Netherton Syndrome....
NANTONG and SUZHOU, China — Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-18, one of the company’s lead saRNA product candidates. This designation follows the recent Rare Pediatric Disease...
HOUSTON, Texas & PARIS, France — RadioMedix, Inc. and Orano Med, two clinical stage radiopharmaceutical companies, today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to AlphaMedixTM (212Pb-DOTAMTATE) for the treatment of adult patients with unresectable or metastatic, progressive somatostatin receptor expressing...
NEW HAVEN, Conn. — Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases, today announced a collaboration with Johnson & Johnson to support the development of complementary therapeutic approaches aimed at reducing the risk...
NEW HAVEN, Conn. — Rallybio Corporation announced preliminary Phase 1 multiple ascending dose (MAD) data for RLYB116, an innovative, long-acting, low volume subcutaneously injected inhibitor of complement component 5 (C5), in development for patients with complement-mediated diseases. The Phase 1 MAD study for RLYB116 evaluated the safety, tolerability, pharmacokinetics (PK),...
NOVATO, California — The Phase 2b clinical trial will enroll up to six cystinosis patientswith a history of good compliance using the currently availableimmediate-release form of cysteamine bitartrate, the only drug cleared formarketing by the U.S. Food and Drug Administration (“FDA”) and EuropeanMedicines Agency (“EMEA”) to treat cystinosis. The clinical...
There are more than 7,000 rare diseases identified in the world today. While “rare” means that less than 200,000 people in the US are affected, to suffer from a rare disease is not actually rare at all. Around 10% of the American population suffers from a rare disease. A shocking...
HRA Pharma Rare Diseases and Celnova Pharma are partnering to meet a major unmet need for rare disease drugs in South America.