Makena's FDA approval sparks applause, ire, then dialogue

When the US Food and Drug Administration approved Makena, a branded formulation of the synthetic progesterone 17 alpha-hydroxyprogesterone caproate (17OHP) on February 4, obstetricians and maternal-fetal medicine specialists celebrated a perceived victory in the fight to reduce the number of premature births: Because 17OHP previously was available only through a limited number of compounding pharmacies, approval of the drug theoretically would make a standardized product available to more women at high risk for premature delivery.

However, since then, KV Pharmaceuticals (St. Louis, Missouri), the manufacturer of Makena, announced that FDA had granted it sole production rights to the drug and that the cost of a single dose now would be $1,500 instead of the $10 to $30 charged for the drug compounded by pharmacists, bringing the cost of a 20-week course of treatment to approximately $30,000, excluding pharmacy costs and markups. KV also sent a letter threatening legal action against pharmacies continuing to compound 17OHP.

Clinicians were outraged. Of 4.2 million live births in the US each year, 1 in 8 is premature, and more than $25 billion is spent annually for the immediate and long-term care of children born prematurely. Although KV has said it will offer financial assistance to eligible women, health insurance premiums are expected to increase, and uninsured women who are ineligible for KV's assistance program would likely be unable to expeditiously begin 17OHP treatment while their application and qualification for free or discounted drug claims are processed by the manufacturer.

Ther-Rx has issued a statement: "We recognize the concerns that have been raised regarding the list price, patient access, and potential cost to payors of this important orphan drug... . The company is scheduling meetings with key audiences including payors and national organizations that are committed to the advancement of obstetric care and infant health." Greg Divis, president of Ther-Rx, has agreed to meet with Dr Howse and leaders from ACOG, SMFM, and AAP to discuss the pricing structure of Makena and the patient assistance program.