To clarify the extent of patent protection and the right to benefit from the orphan incentive package, the European Commission (DG24 committee) recently defined "similarity" between orphan products
as the same substance, or a substance that differs from the original substance in molecular structure, source material, or manufacturing process, or an organism (living or nonliving) that is comparable with the original substance or organism in terms of biologic action and properties (including efficacy and safety) and ability to act through the same mechanism.
Marlene Haffner, director of the Office of Orphan Products
Development, credits the Orphan Drug Act for summoning major pharmaceutical concerns to the aid of the many small groups of people afflicted with rare, and often untreatable, diseases.
MeiraGTx announced that the Offices of Orphan Products
Development and Pediatric Therapeutics of the FDA have granted rare pediatric disease designation to the company's gene therapy product candidate AAV-CNGA3 for the treatment of patients with achromatopsia, or ACHM, due to mutations in the CNGA3 gene.
Medicine company NeuroVive Pharmaceutical AB (Nasdaq Stockholm:NVP) (OTCQX:NEVPF) reported on Wednesday the receipt of the Orphan Drug Designation from the US FDA Office of Orphan Products
Development for KL1333 for treatment of inherited mitochondrial respiratory chain diseases (MRCD).
Thalidomide, the drug that crippled hundreds of babies before they were born because their mothers took it to control nausea, and provoked the development of modern drug approval processes in the 1960s, is the most notable of the candidates for further research that have just won EU designation as orphan products
. The application has been submitted by Laphal of France, which is studying the compound for treatment of erythema nodosum leprosum.
Orphan designation is granted by the FDA Office of Orphan Products
Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S.
Biopharmaceutical company Stealth BioTherapeutics said on Monday that the US Food and Drug Administration (FDA) Office of Orphan Products
Development has awarded orphan drug designation for its investigational drug candidate, elamipretide, for the treatment of patients with Barth syndrome.
Food and Drug Administration Office of Orphan Products
Development has granted Orphan Drug Designation to CLR 131, the company's lead Phospholipid Drug Conjugate product candidate, for the treatment of rhabdomyosarcoma, a rare pediatric cancer.