FDA Yanks Makena & Generics off Shelves: Preterm-Birth Drug Deemed Ineffective after 12-Year Reign

The US Food and Drug Administration (FDA) has announced that it has reached a final decision to withdraw approval for Makena, a drug used to prevent preterm birth, and its generic counterparts. The decision comes 12 years after the drug was first approved and made available on the market.

Makena was approved in 2011 using the FDA’s accelerated-approval pathway. This means that the drug was approved on the basis of limited clinical data and, in such cases, manufacturers must conduct post-approval studies to confirm the drug’s efficacy. Makena’s approval came as a treatment designed to reduce the risk of spontaneous preterm birth in pregnant women who already had a history of the condition.

The withdrawal of approval for Makena and its generic versions comes after a series of studies and consultations with external experts. In October 2019, the FDA’s Bone, Reproductive, and Urologic Drugs Advisory Committee evaluated the results of a confirmatory trial conducted by AMAG Pharmaceuticals, the company that manufactures Makena. The trial, which was required as part of the accelerated-approval process, failed to demonstrate the drug’s efficacy in reducing the risk of recurrent preterm birth.

However, there was one trial that showed a reduction in the risk of preterm birth. In this study, patients were randomly assigned to receive either Makena or a placebo. The results indicated that the rate of preterm birth was significantly lower in the Makena group than in the placebo group. Still, the evidence was not strong enough to outweigh the results of the confirmatory trial, which showed no significant differences in the rates of preterm birth among those who received Makena and those who did not.

In February 2020, AMAG Pharmaceuticals announced plans to discontinue Makena, citing a lack of demand for the drug and inadequate reimbursement from insurers. In April 2020, the company sold the rights to the drug to Covis Pharma. However, the FDA continued to review Makena’s safety and efficacy, ultimately leading to the decision to withdraw approval.

The FDA’s decision to withdraw approval for Makena and its generics comes after considering the totality of the evidence, including the outcomes of the confirmatory trial, other available data, and the opinions of external experts. The agency concluded that “there is no demonstration of a clinical benefit for the intended use of Makena.”

Pulling Makena and its generic versions from the market is an unfortunate outcome for patients who relied on the drug to reduce the risk of preterm birth. The FDA’s decision has sparked questions about the accelerated-approval pathway for drugs, as it allowed Makena to be approved on the basis of limited clinical evidence.

The FDA maintains that the accelerated-approval pathway serves a necessary purpose for patients with no other treatment options. However, this case highlights the need for thorough post-approval studies and the importance of the FDA following through on its commitment to monitor drug safety and efficacy even after a product has gained market approval.

It is also a reminder that drug development is a complex process, with potential setbacks at any stage. In this case, the withdrawal of Makena’s approval marks the end of its journey, but its story serves as a cautionary tale for other drugs in development, reminding manufacturers and regulators of the importance of thorough scientific investigation and the need for robust evidence to support the safety and efficacy of a drug.

In conclusion, the FDA’s decision to withdraw approval for Makena and its generics is a significant development that has implications for the drug development process as a whole. This case illustrates the importance of the accelerated-approval pathway for providing patients with treatment options when there are no alternatives, but also underscores the need for thorough post-approval studies and ongoing monitoring of drug safety and efficacy. As Makena’s story comes to an end, it is crucial for manufacturers and regulators to learn from its experience and ensure that future drugs are supported by robust evidence and thorough scientific investigation.


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