FDA Grants Orphan Status To Arca's Ebola Treatment, Insys' Gastric Cancer Treatment
By Cyndi Root
The Food and Drug Administration (FDA) has granted Orphan Drug status to an Ebola treatment and a gastric cancer treatment. Arca Biopharma’s rNAPc2 is a drug for viral hemorrhagic fever after exposure to the Ebola virus.
Insys was granted orphan status for Liposome Entrapped Paclitaxel Easy to Use (LEP-ETU) an improved formulation of paclitaxel to treat gastric cancer. Orphan drug designation confers seven years of market exclusivity, fee waivers, and other benefits to the sponsor upon approval.
rNAPc2
rNAPc2 (recombinant nematode anticoagulant protein c2) is a candidate from Arca Biopharma, a Westminster, Colorado-based company. The drug is a selective inhibitor of tissue factor (TF), a protein that initiates the primary coagulation mechanism. rNAPc2 was originally developed as a cardiovascular therapy. The agent has shown effectiveness against Ebola in animal models. Arca states that it is seeking development partners, out-licensing, and government funding to move the agent through the development process.
Arca BioPharma announced in August 2014 that Health Canada accepted its Clinical Trial Application (CTA) for the GENETIC-AF clinical trial investigating Gencaro for the treatment of atrial fibrillation (AF). The company expects clinical sites in Canada to be active in Q4 2014 for the Phase 2b/3 trial. Arca states that personalized medicine is its focus in the trial, as Gencaro has potential as a genetically-targeted therapy.
LEP-ETU
LEP-ETU is Insys Therapeutics’ agent to treat gastric cancer, acquired from NeoPharm during a merger in 2010. The company has reformulated paclitaxel by entrapping the drug with liposomes, which has helped boost efficacy and reduce toxicity. Michael L. Babich, president and CEO of Insys, stated that his company is evaluating the next steps to take with the drug product candidate.
In November 2014, Insys announced that it received orphan drug status from the FDA for its pharmaceutical cannabidiol (CBD) candidate to treat pediatric schizophrenia. The company expects to dose its first patient in a Phase 1 clinical trial starting in early 2015. In October 2015, Insys updated its progress with its Dronabinol Oral Solution, stating that the FDA requires additional information on the pediatric study plan. The company says that it can meet the FDA’s requirement and resubmit the application without further clinical trials. The company seeks to use the agent for anorexia, AIDS, and cancer chemotherapy-induced nausea and vomiting.