In 2010, the FDA approved the prescription blood-thinning drug Pradaxa (dabigatran) to prevent stroke and systemic blood clots in patients with atrial fibrillation, as well as for the treatment and prevention of deep venous thrombosis and pulmonary embolism.
On October 16, 2015, the FDA granted accelerated approval for the first reversal agent specifically for Pradaxa, called Praxbind ((idarucizumab), which is a solution for intravenous injection during emergency situations when there is a need to reverse Pradaxa’s blood-thinning effects. Praxbind works by binding to the drug compound to neutralize its effect. Both Praxbind and Pradaxa are marketed by Boehringer Ingelheim of Ridgefield, Connecticut.
The FDA cautioned that reversing the effect of Pradaxa exposes patients to the risk of blood clots and stroke from their underlying disease, such as atrial fibrillation. The Praxbind labeling recommends patients resume their anticoagulant therapy as soon as medically appropriate, as determined by their health care provider.
Praxbind was approved by the FDA under its accelerated approval program, which allows the FDA to approve drugs for serious conditions that fill an unmet medical need based on an effect on a surrogate or an intermediate clinical endpoint that is reasonably likely to predict a clinical benefit to patients. The program is designed to provide patients with earlier access to promising new drugs, but the pharmaceutical company will be required to submit additional clinical information after approval to confirm the drug’s clinical benefit.
The FDA reported that the safety and effectiveness of Praxbind were studied in three trials involving a total of 283 healthy volunteers taking Pradaxa, who did not require an anticoagulant. In the healthy volunteers who were given Praxbind, there was an immediate reduction in the amount of Pradaxa in participants’ blood as measured by unbound dabigatran plasma concentration, which lasted for at least 24 hours. Headache was the most common side effect from use of Praxbind in this study.
In 123 patients taking Pradaxa in an ongoing trial who received Praxbind due to uncontrolled bleeding or because they required emergency surgery, the anticoagulant effect of Pradaxa was fully reversed in 89% of patients within four hours of receiving Praxbind. In this patient trial, the most common side effects were low potassium (hypokalemia), confusion, constipation, fever, and pneumonia.
FDA’S Accelerated Approval Program
In 1992, the FDA promulgated regulations for its accelerated approval program that allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint (a surrogate endpoint used for accelerated approval is a marker such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit (a clinical benefit is a positive therapeutic effect that is clinically meaningful in the context of a given disease)).
In 2012, the U.S. Congress passed legislation that allows the FDA to base accelerated approval for drugs for serious conditions that fill an unmet medical need on whether the drug has an effect on a surrogate or an intermediate clinical endpoint (an intermediate clinical endpoint is a measure of a therapeutic effect that is considered reasonably likely to predict the clinical benefit of a drug, such as an effect on irreversible morbidity and mortality).
Using surrogate or intermediate clinical endpoints can save valuable time in the drug approval process but the drug manufacturer will still need to conduct phase 4 confirmatory trials. Approval of a drug may be withdrawn or the labeled indication of the drug changed if trials fail to verify clinical benefit or do not demonstrate sufficient clinical benefit to justify the risks associated with the drug.
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