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FDA highlights 2020 rare disease drug approvals

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In an editorial for FDA Voices, Patrizia Cavazzoni, MD, Acting Director, Center for Drug Evaluation and Research (CDER) highlighted a number of new drug therapies that were approved in 2020 for rare diseases, including epithelioid sarcoma.

“New drug therapies for patients with rare diseases are often among the most important approvals. Patients with rare diseases frequently have few or no drugs available to treat their condition — and for them, approvals of so-called ‘orphan’ drugs can mean new hope for an enhanced quality of life, and in some cases, increased survival,” she wrote.

In 2020, the CDER approved 53 novel drugs. More than half of those (31) were new approval for drugs to treat patients with rare diseases.

A number of novel therapies for rare cancers, such as multiple myeloma, neuroblastoma, and mesothelioma, were approved, including epithelioid tazemetostat (Tazverik) for the treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.

Tazemetostat was approved in January 2020.

“Epithelioid sarcoma accounts for less than one percent of all soft tissue sarcomas,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research in a press release announcing the approval. “Until today, there were no treatment options specifically for patients with epithelioid sarcoma. The approval of Tazverik provides a treatment option that specifically targets this disease. When we brought Tazverik’s application to the Oncologic Drugs Advisory Committee last month, the committee voted unanimously that the benefits of the drug outweighed the risks.”

Cavazzoni P. 2020: A Strong Year for New Drug Therapy Approvals – Despite Many COVID-19 Challenges. FDA Voices. Available at: https://www.fda.gov/news-events/fda-voices/2020-strong-year-new-drug-therapy-approvals-despite-many-covid-19-challenges