First Drug for Peripheral T-cell Lymphoma Approved

The FDA has approved Folotyn (pralatrexate), the first treatment for a form of cancer known as peripheral T-cell lymphoma (PTCL), an often aggressive type of non-Hodgkin's lymphoma.
Folotyn was approved under the FDA's accelerated approval process, which allows earlier approval of drugs that meet unmet medical needs. The drug is approved for patients who have relapsed or have not responded well to other forms of chemotherapy.

Lymphoma is a cancer of the lymphatic system, which is part of the immune system. There are many types of lymphoma: one type is called Hodgkin's disease, and the rest are called non-Hodgkin's lymphomas. PTCL involves T-cells, a type of white blood cell. This relatively rare disease occurs in less than 9,500 patients each year in the US.

"Folotyn's approval demonstrates FDA's commitment to the rapid approval of drugs for rare and uncommon diseases," said Richard Pazdur, MD, director of the Office of Oncology Drug Products in the FDA's Center for Drug Evaluation and Research.

When studying a new drug, it can take time to learn whether a drug actually provides real improvement for patients, such as living longer or feeling better. This real improvement is known as a clinical outcome. In 1992, the FDA instituted accelerated approvals that allow earlier approval of drugs based on a surrogate endpoint, a laboratory measurement or physical sign that can serve as an indirect or substitute measurement for clinical outcomes.

In Folotyn's case, the FDA approved the drug based on evidence that it reduces tumor size, because tumor shrinkage is considered reasonably likely to predict a clinical benefit, such as extending the survival of cancer patients. Tumor shrinkage was seen on imaging scans in one study. Of 109 patients with PTCL in the trial, 27 percent had reduction in tumor size.

To speed the drug's availability, Folotyn was granted priority review, ensuring a review within six months rather than 10 months for a standard review. The drug was also designated an orphan drug, which provides a variety of financial incentives to manufacturers that develop drugs for a small number of patients with a rare disorder.

The most common adverse reactions seen with Folotyn were irritation or sores of the mucous membranes (such as the lips, the mouth and the digestive tract), low platelet cell counts, low white blood cell counts, fever, nausea and fatigue.

Folotyn can harm a fetus. Women should avoid becoming pregnant while being treated with this drug, and pregnant women should be informed of the potential risk.

Patients treated with Folotyn should take folate (vitamin B9) and vitamin B12 supplements to reduce mucous membrane irritation.

Folotyn is manufactured by Allos Therapeutics Inc. As a condition of accelerated approval, Allos will conduct studies to confirm that tumor shrinkage actually does predict that patients will live longer.

Contact:
Karen Riley
301-796-4674
karen.riley@fda.hhs.gov