By Anna Rose Welch, associate editor
Horizon Pharma Wins Orphan Drug Designation
Actimmune (interferon gamma-1b) was designated an orphan drug for Friedreichs Ataxia. Actimmune is currently approved to treat infections caused by Chronic Granulomatous Disease (CGD), as well as for severe, malignant osteopetrosis (SMO) that disturbs the bone marrow. There are currently no treatments approved for Friedreichs Ataxia, which would mean the drug, if approved, could meet an unmet health need currently affecting 1 in 50,000 people in the U.S.
EffRx Drug Achieves Orphan Status In PCOS
EffRx drug, EX404, indicated for pediatric polycystic ovary syndrome (PCOS), received orphan drug designation last week. The disorder is believed to originate from a hormonal imbalance that results from increased androgens and/or insulin. Those with the disorder are often at risk for developing diabetes type 2, infertility, heart disease, and other conditions. The company is currently at work planning a clinical development program for the drug candidate, with expectations of launching the candidates first trials sometime this fall.
Spinal Muscular Atrophy Drug Granted Orphan Designation
ChariSMA, also known as scAAV9 from synthetic biology company AveXis, received orphan drug status for Spinal Muscular Atrophy (SMA). The intravenous gene therapy drug is delivered using technology licensed from ReGenX Bioscience and Research Institute at Nationwide Childrens Hospital. The drug is responsible for using a genetically modified virus, which inserts a replacement Survival Motor Neuron (SMN) gene to make up for the lack of SMN production as a result of the malfunctioning SMN1 gene. The SMN gene plays a key role in helping infants breath, sit up, and swallow.
Chimerix Given FDA Ok For Emergency Ebola Treatment
Biopharmaceutical company Chimerix was given the greenlight to distribute its investigational drug candidate, Brincidofovir (CMX001), to patients with Ebola following the FDAs approval of its Emergency Investigational New Drug Applications (EIND). Over the past few years, the drug has been in a Phase 3 program investigating its effect on cytomegalovirus and adenovirus, which gave enough information on the drugs safety and dosing to explore its potential against Ebola. The FDA is working with Chimerix to create a clinical development plan for the drug as a treatment for Ebola. The drug has already been tested at the NIH and CDC, where it demonstrated a similar effect on the Ebola Virus as it did against adenovirus and smallpox.
Eylea Gains Approval For Macular Edema Following RVO
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FDA News Roundup: Chimerix, Horizon, Regeneron, And More