FDA awards more than a dozen grants for rare disease research
SILVER SPRING, Md. — The Food and Drug Administration is offering more than $14 million in grants for the development of products for patients with rare diseases, the agency said Monday.
The FDA announced that it had granted 15 grants for orphan drug research. As defined by the agency for drugs, orphan diseases affect fewer than 200,000 people in the United States. For devices, a rare disease occurs so infrequently in the country that there is no reasonable explanation that a medical device for such a disease will be developed without assistance. According to the National Institutes of Health, there are about 6,800 rare diseases and conditions, affecting nearly 30 million Americans.
Among the grants, Alkeus Pharmaceuticals received $167,000 for a drug for Stargardt disease currently in phase-1 development; Children’s Hospital Medical Center in Cincinnati received $600,000 for a phase-1 study of a drug to treat Fanconi anemia; Children’s Hospital received $600,000 for a phase-1 study of a drug for Wiskott-Aldrich syndrome; and Vanderbilt University received $1.59 million for a phase-2 study of montelukast, which Merck markets as the asthma and allergy drug Singulair, for the treatment of sickle-cell anemia.
"The FDA is committed to fostering and encoring the development of products for rare diseases, most of which have no available or adequate treatments," FDA Office of Orphan Product Development director Gayatri Rao said. "The grants awarded this year support studies in very vulnerable, difficult-to-treat populations who have no available options."
FDA approves Iroko Pharmaceuticals NSAID
PHILADELPHIA — The Food and Drug Administration has approved a pain drug made by Iroko Pharmaceuticals, Iroko said.
The drug maker announced the approval of Zorvolex (diclofenac) capsules for mild to moderate acute pain in adults. The drug is a non-steroidal anti-inflammatory drug, or NSAID, belonging to the same class as the common analgesic drug ibuprofen. Zorvolex was approved at dosage strengths 20% lower than currently available diclofenac products, the company said.
"The approval of Zorvolex is important news for patients and for physicians who need new options for effective pain relief and is a significant milestone for Iroko," Iroko president and CEO John Vavricka said. "This marks a major achievement toward our goal of applying new technology to existing NSAIDs in order to address unmet medical needs in analgesia."
FDA approves Opsumit for pulmonary arterial hypertension
SILVER SPRING, Md. — The Food and Drug Administration has approved a new drug made by Actelion Pharmaceuticals for treating pulmonary arterial hypertension.
The agency announced the approval of Opsumit (macitentan) for adults with PAH, a chronic, progressive and debilitating disease caused by high blood pressure in the arteries that connect the heart to the lungs that can lead to death or the need for lung transplantation.
The FDA said the drug would carry a boxed warning, the highest warning possible, alerting patients and healthcare providers that Opsumit should not be used in pregnant women because it can harm a developing fetus. Women can only receive the drug through a risk evaluation and mitigation strategy program.