NOVATO, Calif., July 22, 2013 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. (Nasdaq:RPTP) today announced that it has received a positive opinion from the European Union (EU) Committee for Orphan Medicinal Products (COMP) recommending orphan drug exclusivity for PROCYSBI ® (mercaptamine) gastro-resistant hard capsules for the treatment of proven nephropathic cystinosis. Final adoption of the opinions on drug approval and orphan exclusivity are expected from the European Commission in the coming months.
"The positive opinion from COMP is another welcome milestone for Raptor and PROCYSBI as we seek to bring this important new product to European patients who suffer from cystinosis," said Christopher M. Starr, Ph.D., Raptor's chief executive officer. "Orphan designation will provide the company with 10 years of marketing exclusivity in the European Union alongside our IP estate."
The EMA's Orphan Drug Exclusivity is designed to provide regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or debilitating conditions with a prevalence in the European Union (EU) of not more than five in 10,000. Orphan medicinal products receive 10 years of market exclusivity in the EU after receiving marketing authorization.U.S. Product Information about PROCYSBI (cysteamine bitartrate) PROCYSBI is a cystine-depleting agent that is approved in the U.S. for the management of nephropathic cystinosis in adults and children ages 6 years and older. It is contraindicated in patients with a hypersensitivity to penicillamine. The most commonly reported side effects are vomiting, abdominal pain/discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite, breath odor, fatigue, dizziness, skin odor, and rash. If European Commission approval is obtained, the indications, contraindications, warnings and precautions ultimately adopted by the European Commission may be different from those in the U.S. Important U.S. Safety Information about PROCYSBI
- Patients should be monitored for development of skin or bone lesions and dosage of PROCYSBI reduced as needed.
- If a severe skin rash develops such as erythema multiforme bullosa or toxic epidermal necrolysis, PROCYSBI should be discontinued.
- Cysteamine has been associated with gastrointestinal ulceration and bleeding.
- Central Nervous System (CNS) symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine. Patients should exercise caution in driving a car or engaging in other hazardous activities after taking PROCYSBI.
- Cysteamine has been associated with reversible leukopenia and abnormal liver function studies. Therefore, blood counts and liver function tests should be monitored.
- Benign intracranial hypertension (or pseudotumor cerebri PTC) and/or papilledema has been associated with immediate-release cysteamine bitartrate treatment. Physicians should monitor for signs and symptoms of PTC.
- Breastfeeding is not recommended for nursing mothers taking PROCYSBI.