The Food and Drug Administration said Feb. 4 that it had approved 17-alpha-hydroxyprogesterone caproate, a progesterone injection also known as 17P, for the prevention of recurrent preterm birth in women with singleton pregnancies.
The FDA said in a press statement that it had approved 17P (Makena) under the agency’s accelerated approval regulations that allow drugs to be approved based on a surrogate end-point benefit that is "reasonably likely to predict a clinical benefit."
Hydroxyprogesterone caproate was initially approved in 1956 for treatment and prevention of recurrent miscarriage, among other indications, but was withdrawn in 2000 because of manufacturing problems. Hospital pharmacies continued to mix the compound, and it remained available in many settings.
The March of Dimes said in a statement Feb. 4 that it welcomed 17P’s approval. "Prior to today’s approval of Makena, health care providers ordered prescriptions of 17P from compounding pharmacies; however, many eligible patients faced logistical and financial barriers to access. FDA approval means the drug now will be widely available."
The drug was first marketed in the 1950s as Delalutin. It was resubmitted to the FDA in 2006 as Gestiva by a California firm, and will now be marketed by Ther-Rx Corp., K-V Pharmaceutical’s branded drug subsidiary, as Makena.*
The approval comes nearly 5 years after an FDA advisory panel voted in 17P’s favor, based on results from a publicly funded, randomized, double-blind placebo-controlled trial of 463 women with at least 1 preterm delivery (N. Engl. J. Med. 2003;348:2379-85). That trial showed that women receiving weekly injections of 250 mg of 17P, starting at 20 weeks and continuing to 36 weeks or delivery, saw a 24% reduced risk of delivery before 37 weeks compared with the control group. The study also showed reductions in the rates of preterm delivery before 35 weeks and before 32 weeks in the treatment arm.
After the FDA panel’s vote in August 2006, the agency demanded further safety studies, including now-completed studies of children born to women taking 17P, from the medicine’s then-developer, which later sold its rights to 17P. In 2010 the drug’s current developer, Hologic, resubmitted its filing for 17P.
The FDA said Feb 4 that the drug’s approval comes with the manufacturer’s responsibility to complete ongoing confirmatory studies, including a new infant follow-up study, expected to end in 2018 and to include between 580 and 750 infants.
* CORRECTION, 2/17/2011: The original version of this article incorrectly stated that Makena will be marketed by Hologic, Inc. The drug will instead be marketed by Ther-Rx Corp., K-V Pharmaceutical’s branded drug subsidiary. Rights to Makena were transferred from Hologic, Inc. to K-V after the FDA approved the drug.