|GW Pharma Receives Orphan Drug Designation by FDA for Epidiolex(R); OPKO's VIIa-CTP Receives Orphan Drug Designation|
|By Staff and Wire Reports|
|Friday, 28 February 2014 20:13|
GW Pharmaceuticals (Nasdaq: GWPH) announced the U.S. FDA has granted orphan drug designation for Epidiolex®, GW's product candidate that contains plant-derived Cannabidiol (CBD) as its active ingredient, for use in treating children with Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood-onset epilepsy. Epidiolex is an oral liquid formulation of a highly purified extract of CBD, a non-psychoactive molecule from the cannabis plant.
In November 2013, GW announced that the FDA had granted orphan drug designation for Epidiolex in the treatment of Dravet syndrome. Following the successful follow-on offering completed in January 2014, GW intends to advance a full clinical development program for Epidiolex for the treatment of both LGS and Dravet syndromes. Working with leading pediatric epilepsy specialists in the United States, GW expects to commence an initial Phase 2 clinical trial for Epidiolex in the second half of 2014. This trial, if successful, is expected to lead to Phase 3 pivotal trial programs in both Dravet syndrome and LGS intended to support New Drug Applications with the FDA.
LGS is a severe form of childhood-onset epilepsy. Seizure types, which vary among patients, include tonic (stiffening of the body, upward deviation of the eyes, dilation of the pupils, and altered respiratory patterns), atonic (brief loss of muscle tone and consciousness, causing abrupt falls), atypical absence (staring spells), and myoclonic (sudden muscle jerks). Most children with LGS experience some degree of impaired intellectual functioning or information processing, along with developmental delays, and behavioral disturbances. LGS can be caused by brain malformations, perinatal asphyxia, severe head injury, central nervous system infection and inherited degenerative or metabolic conditions. It is estimated that there are approximately 14,000-18,500 patients with LGS in the United States and 23,000-31,000 patients with LGS in Europe.
"This orphan designation for Epidiolex, GW's purified CBD medicine, in Lennox-Gastaut syndrome follows on from the recent FDA grant of orphan designation in Dravet syndrome. We are now in active discussions with the FDA regarding the US regulatory pathway for Epidiolex and believe that this medicine has the potential to meet the significant unmet need of children suffering with severe seizures where all options to control those seizures have been exhausted," stated Justin Gover, GW's Chief Executive Officer. "GW is responding to this need with the goal of providing an FDA-approved prescription CBD medicine that physicians have confidence in prescribing and parents can trust for quality, consistency and access."
OPKO Health, Inc. (NYSE:OPK) announced the U.S. FDA has granted orphan drug designation to OPKO's longer-acting version of clotting Factor VIIa (Factor VIIa-CTP) for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors to Factor VIII or Factor IX. These patients are currently being treated by commercially-available Factor VIIa, with estimated 2013 worldwide sales of $1.7 billion.
Currently, Factor VIIa therapy is available only as an intravenous (IV) formulation, which requires multiple injections to treat a bleeding episode due to Factor VIIa’s short half-life. In addition, the requirement for multiple weekly injections can be onerous for patients interested in preventive prophylactic treatment of the disease, especially children.
Preclinical data presented by OPKO at the 7th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) on February 26-28, 2014 in Brussels show that OPKO’s long-acting Factor VIIa-CTP demonstrated the potential for substantial improvement of the quality of life of patients, via both IV and subcutaneous (SC) administration. The combination of a long-acting Factor VIIa coupled with SC administration using a simple injection could potentially change the Factor VIIa market segment, allowing children and adults with hemophilia to easily self-administer the drug at home on a prophylactic basis.
Dr. Phillip Frost, CEO of OPKO, commented, “We are pleased with the new orphan drug designation for Factor VIIa-CTP in the U.S., as it would allow OPKO seven years of marketing exclusivity upon commercialization of the drug. We have previously presented data in animal models of hemophilia showing that Factor Vlla-CTP demonstrates superiority across key efficacy and safety parameters compared to current Factor VIIa therapy. We are enthused about the potential for SC administration due to its improved bioavailability compared to presently available Factor VIIa IV therapy. Expecting to initiate a Phase IIa trial with our IV formulation in hemophilia patients in late 2014, we believe that we are in a unique position to be first to market with a longer acting Factor VIIa product.”
FDA's Orphan Drug Act of 1983 was designed to encourage the development of products that demonstrate promise for the diagnosis, prevention and/or treatment of life-threatening or very serious conditions that are rare and affect 200,000 persons or less in the U.S. The orphan drug designation provides for seven years of market exclusivity, reduction in regulatory fees, certain tax credits and additional regulatory support for R&D initiatives.
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