Is jakafi an orphan drug?
JAKAFI is an orphan drug approved to treat patients with: (1) intermediate or high-risk myelofibrosis; (2) polycythemia vera in patients who have had an inadequate response to or are intolerant of hydroxyurea; and (3) steroid-refractory acute graft-versus-host disease.
What did the orphan drug law allow for?
ch. 9 § 301 et seq. The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington’s disease, myoclonus, ALS, Tourette syndrome and muscular dystrophy which affect small numbers of individuals residing in the United States.
Who can dispense jakafi?
Jakafi is one of many medicines available only through specialty pharmacies. You can’t fill your prescription for it at a traditional local pharmacy.
How long does it take to get an orphan drug approved?
The FDA will review and make a decision within 60 days from the request.
How do you qualify for orphan drug designation?
Criteria for Orphan Drug Designation Adequate documentation or prevalence data must demonstrate that the intended condition is rare. There must be a scientific rationale establishing a medically plausible basis for the use of the product for the rare condition.
How many orphan drugs have been approved?
In 2020, we continued to see significant progress in the development of treatments for rare diseases, also known as orphan products. Specifically, in 2020, the agency approved 32 novel drugs and biologics with orphan drug designation.
Why has the Orphan Drug Act been successful?
Many researchers and policy makers have credited the Orphan Drug Act—in particular, its guarantee of seven years of market exclusivity—with this success, citing the surge in drugs for rare diseases as evidence that legislative grants of market exclusivity work to generate pharmaceutical innovation.
Is jakafi a specialty drug?
What Is a Specialty Pharmacy? Specialty pharmacies usually handle medicines that require more care or that are prescribed for less common or more complex health conditions. Jakafi is one of many medicines available only through specialty pharmacies.