FDA News Roundup: Chimerix, Horizon, Regeneron, And More

Horizon Pharma Wins Orphan Drug Designation

Actimmune (interferon gamma-1b) was designated an orphan drug for Friedreich’s 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 Friedreich’s 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 candidate’s 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 Children’s 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 FDA’s 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 drug’s 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

Eylea received yet another approval from the FDA, this time for the treatment of Macular Edema following Retinal Vein Occlusion (RVO). The drug was recently approved for the treatment of macular edema following Central Retinal Vein Occlusion (CRVO). Patients have been instructed to take 2 mg of the drug over 4 weeks in this new indication. In trials, the drug helped patients regain at least 15 letters in vision on an eye chart. Overall, patients saw a 17-letter mean improvement in best-corrected visual acuity over baseline compared to those in the control group who received macular laser photocoagulation and who only averaged a 6.9 percent mean improvement.

Vivolux Cleared For Phase 1 Trial

The FDA has granted Vivolux the permission to proceed with a Phase 1/2 clinical trial for its multiple myeloma drug VLX1570. The drug will be investigated for safety and efficacy in a trial carried out with the Memorial Sloan-Kettering Cancer Center, Dana Farber Cancer Institute, and Harvard Medical School.  As resistance to proteasome inhibitors (i.e. Velcade) in the treatment of multiple myeloma continues to increase, researchers hope this drug will offer a different mechanism of action that causes cancer cell apoptosis.

DNATrix Gains Orphan Drug Designation For Glioma Treatment

DNATrix’s treatment for glioma, DNX-2401, was awarded an orphan drug designation. The drug, a conditionally-replicative oncolytic adenovirus for malignant glioma, uses a live virus to invade and multiply in cancer cells. In doing so, healthy tissues are spared while the virus spreads from tumor cell to tumor cell, resulting in tumor death and potential long-term survival for patients. The drug, which received Fast-Track designation from the FDA, is currently in clinical trials in the U.S. and Europe.

Brain Storm Cell Therapeutics Put On Fast-Track By FDA

NurOwn was granted Fast-Track Designation as a treatment for amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s Disease). The drug, which is comprised of autologous mesenchymal stem cells that secrete neurotrophic factors, is being investigated in a phase 2 ALS trial. There are currently 30 patients taking NurOwn in an ongoing trial in Israel and in the U.S.