Veralox Therapeutics Announces FDA Orphan Drug Designation for VLX-1005
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The U.S. Food and Drug Administration has granted Orphan Drug Designation for VLX-1005 for the treatment of heparin induced thrombocytopenia.
“This is a significant regulatory milestone for our company, and in the development of this investigational new drug product and recognition of the clear unmet medical need for patients who suffer from this devastating disease.” - Jeffrey W. Strovel, PhD., CEO, Veralox Therapeutics FREDERICK, Md. (PRWEB) January 27, 2021 Veralox Therapeutics, a biotechnology company developing first-in-class small molecule therapeutics that treat the underlying pathologies of diseases with significant unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for VLX-1005 for the treatment of heparin induced thrombocytopenia (HIT).
Published: Jan 19, 2021
DALLAS (BUSINESS WIRE) Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received both rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.
“There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease,” said RA Session II, President, Founder and CEO of Taysha. “We are encouraged by the early evidence of TSHA-105’s disease-modifying approach and believe these designations will help us potentially accelerate the development of this exciting program. We look forward to working with the FDA to make TSHA-105 available to patient
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Istari Oncology Announces FDA Grants Orphan Drug Designation for PVSRIPO for the Treatment of Advanced Melanoma
Istari Oncology Announces FDA Grants Orphan Drug Designation for PVSRIPO for the Treatment of Advanced Melanoma Istari Oncology, Inc., a clinical-stage biotechnology company, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for PVSRIPO for the treatment of advanced melanoma (stage IIB-IV). PVSRIPO is a novel viral immunotherapy, based on the Sabin type 1 polio vaccine, that activates a patients innate and adaptive immune system to facilitate an anti-tumor response and establish long-term immunologic memory to help prevent the cancers return.
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Amryt Granted Orphan Drug Designation by the FDA for AP103
Amryt Pharma plcDecember 23, 2020 GMT
Amryt Granted Orphan Drug Designation by the FDA for AP103
DUBLIN, Ireland, and Boston MA, December 23, 2020, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, is pleased to announce today that the U.S. Food and Drug Administration (“FDA”) has granted orphan drug designation for AP103 for the treatment of Dystrophic Epidermolysis Bullosa (“DEB”). AP103 is based on Amryt’s gene-therapy platform technology and offers a potential treatment for patients with DEB, a subset of EB.
Sigilon Therapeutics Receives Orphan Drug Designation for SIG-005 for the Treatment of Mucopolysaccharidosis Type I globenewswire.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from globenewswire.com Daily Mail and Mail on Sunday newspapers.