February 8, 2019 - FDA Approvals, News & Updates

News and Updates —February 1, 2019

This week, the US Food and Drug Administration (FDA) approved a new drug for a rare blood clotting disorder and alerted healthcare providers to a cancer risk associated with breast implants.

In This Article


FDA Approves First Therapy for the Treatment of Adult Patients with a Rare Blood Clotting Disorder

On February 6, 2019, the FDA approved caplacizumab-yhdp (Cablivi; Ablynx), the first drug therapy approved for adults with acquired thrombotic thrombocytopenic purpura (aTTP). The drug received an FDA Fast Track designation and was evaluated under Priority Review, which is reserved for medications that represent significant improvements in safety or efficacy in treating serious conditions.

aTTP is a rare and life-threatening disorder that patients may develop because of various medical conditions, including cancer, HIV, pregnancy, infections, or lupus; they are also at higher risk for the disorder following surgery, transplantation, or chemotherapy. aTTP causes the formation of extensive clots throughout the body, which can block the delivery of blood and oxygen to the organs, resulting in heart attack or stroke. When not treated promptly, the condition is usually fatal.

Caplacizumab-yhdp, which is administered by injection, is used in combination with plasma exchange and immunosuppressive therapy to treat aTTP. “Cablivi is the first targeted treatment that inhibits the formation of blood clots. It provides a new treatment option for patients that may reduce recurrences,” said Richard Pazdur, MD, director of the FDA Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research.

The efficacy of caplacizumab-yhdp was assessed in a phase 3 clinical trial of 145 patients who were randomized to receive either caplacizumab-yhdp (n = 72) or placebo (n = 73). Patients in both groups received the current standard of care of plasma exchange and immunosuppressive therapy. The results of the trial demonstrated that platelet counts improved faster among patients in the caplacizumab-yhdp arm compared with those in the placebo arm. There was also a lower total number of patients with aTTP-related death or recurrence of aTTP during the treatment period with caplacizumab-yhdp.

In clinical trials, frequently reported side effects included bleeding of the nose and gums and headache. Healthcare providers should be aware of a warning in the drug’s prescribing information regarding the risk for severe bleeding.

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FDA Issues Letter to Healthcare Providers Alerting Them to a Risk for Breast Implant Associated–Anaplastic Large-Cell Lymphoma

On February 6, 2019, the FDA notified healthcare providers about an association between all breast implants and breast implant associated–anaplastic large-cell lymphoma (BIA-ALCL).

The FDA stated that patients with breast implants have an increased risk for developing BIA-ALCL, and urged all providers to be aware of the possibility of the disease—particularly in patients with new swelling, lumps, or pain around their breast implant—and to expedite the diagnosis of the malignancy.

The recommendation is based on the findings of a more thorough and comprehensive analysis recently conducted by the FDA and reflects the evolving understanding of BIA-ALCL. As shown in an updated study of medical device reports related to ALCL that the FDA has received since 2010, there were 660 cases of BIA-ALCL in the United States. Of those, 457 were unique cases with a total of 9 patient deaths.

BIA-ALCL is a type of lymphoma; it is not breast cancer. When breast implants are inserted into the body during surgery, they are placed behind the breast tissue under the chest muscle. A fibrous scar called a capsule develops around the implant, separating it from the rest of the breast. BIA-ALCL is typically found contained in the fibrous capsule.

In fulfillment of its mission to educate the public, the FDA also issued a statement on its website directed to patients. Signed by Binita Ashar, MD, of the FDA Center for Devices and Radiological Health, it noted the agency’s continuing efforts to inform patients of the health risk from breast implants, including deaths. Dr Ashar also acknowledged that there is much room for misinterpretation and urged women to seek advice from their healthcare providers in evaluating personal risks.

Although the number of cases of BIA-ALCL is small relative to the estimated 1.5 million patients around the world who receive breast implants each year, it is important for healthcare providers and patients to be aware of the FDA’s recent recommendations.

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Last modified: February 11, 2019

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