New Oncology Drugs Available for Rare Cancer Patients

The approval requires that pembrolizumab be given as monotherapy before surgery, in combination with radiation therapy and/or chemotherapy for several weeks after surgery, and as monotherapy thereafter up to two years or until unacceptable toxicity or disease progression. The approval was based on phase III clinical trial results first reported at the AACR Annual Meeting 2025 by Ravindra Uppaluri, MD, PhD, of the Brigham and Women’s Hospital, the Dana-Farber Brigham Cancer Center, and Harvard Medical School.

From April 1 to June 30, 2025, the FDA issued 13 new oncology approvals, expanding treatment options for patients with a variety of cancers. Notably, these approvals included several therapeutics new to the market, as well as new treatment options for patients with rare diseases.

Among these, perioperative pembrolizumab (Keytruda) was approved for the first-line treatment of adult patients with resectable, locally advanced, PD-L1-positive head and neck squamous cell carcinoma (HNSCC). This marked the first time in decades that a new treatment option became available for locally advanced head and neck cancer.

The approval requires that pembrolizumab be given as monotherapy before surgery, in combination with radiation therapy and/or chemotherapy for several weeks after surgery, and as monotherapy thereafter up to two years or until unacceptable toxicity or disease progression. The approval was based on phase III clinical trial results first reported at the AACR Annual Meeting 2025 by Ravindra Uppaluri, MD, PhD, of the Brigham and Women’s Hospital, the Dana-Farber Brigham Cancer Center, and Harvard Medical School.

The Oncology Center of Excellence (OCE) Rare Cancers Program at the FDA promotes the development of safe and effective new drugs and biologics to treat patients with rare cancers. Although there is no universally accepted definition for a rare cancer, the Orphan Drug Act defines a rare disease or condition as one which affects less than 200,000 people in the United States. The National Institute of Health defines rare cancers as those that affect fewer than 40,000 people in the U.S., and many cancer types are even rarer.

From April 1 to June 30, 2025, the FDA issued 13 new oncology approvals, expanding treatment options for patients with a variety of cancers. Notably, these approvals included several therapeutics new to the market, as well as new treatment options for patients with rare diseases.

Among these, perioperative pembrolizumab (Keytruda) was approved for the first-line treatment of adult patients with resectable, locally advanced, PD-L1-positive head and neck squamous cell carcinoma (HNSCC). This marked the first time in decades that a new treatment option became available for locally advanced head and neck cancer.

The approval requires that pembrolizumab be given as monotherapy before surgery, in combination with radiation therapy and/or chemotherapy for several weeks after surgery, and as monotherapy thereafter up to two years or until unacceptable toxicity or disease progression. The approval was based on phase III clinical trial results first reported at the AACR Annual Meeting 2025 by Ravindra Uppaluri, MD, PhD, of the Brigham and Women’s Hospital, the Dana-Farber Brigham Cancer Center, and Harvard Medical School.

The Oncology Center of Excellence (OCE) Rare Cancers Program at the FDA promotes the development of safe and effective new drugs and biologics to treat patients with rare cancers. Although there is no universally accepted definition for a rare cancer, the Orphan Drug Act defines a rare disease or condition as one which affects less than 200,000 people in the United States. The National Institute of Health defines rare cancers as those that affect fewer than 40,000 people in the U.S., and many cancer types are even rarer.

Oncology drugs already available are benefiting patients with rare cancers and no therapeutic alternatives, according to recent reporting from iSanidad.

The U.S. Food and Drug Administration (FDA) approved 10 cancer drugs in 2025 based on clinical trials in which Memorial Sloan Kettering Cancer Center (MSK) played a pivotal role, as reported in MSK-led research. These approvals spanned therapies for a range of solid tumors and blood cancers and included treatments for both common and rare types of tumors.

Among the approvals, vimseltinib (RomvimzaTM) was approved on February 14, 2025, for adults with a rare condition called tenosynovial giant cell tumor (TGCT), also known as pigmented villonodular synovitis. This approval was based on an international phase 3 trial led by William Tap, MD, Chief of the Sarcoma Medical Oncology Service at MSK.

Vimseltinib is a type of targeted therapy called a kinase inhibitor. TGCT is a tumor of the tissue that lines the joints.

the FDA expanded the approval on March 28, 2025, for 177Lu-PSMA-617 (Pluvicto®) so it can be used earlier in treatment for patients with metastatic prostate cancer that has stopped responding to hormone treatment. Previously, the drug was approved only for patients with metastatic cancer that had stopped responding to both hormone therapy and taxane-based chemotherapy.

The FDA uses the term “accelerated approval” for drugs that treat diseases lacking effective treatments. These criteria are slightly different from a standard approval. The term “full approval” applies to drugs that had previously been granted “conditional approval.”

From April 1 to June 30, 2025, the FDA issued 13 new oncology approvals, expanding treatment options for patients with a variety of cancers. Notably, these approvals included several therapeutics new to the market, as well as new treatment options for patients with rare diseases.

Among these, perioperative pembrolizumab (Keytruda) was approved for the first-line treatment of adult patients with resectable, locally advanced, PD-L1-positive head and neck squamous cell carcinoma (HNSCC). This marked the first time in decades that a new treatment option became available for locally advanced head and neck cancer.

The approval requires that pembrolizumab be given as monotherapy before surgery, in combination with radiation therapy and/or chemotherapy for several weeks after surgery, and as monotherapy thereafter up to two years or until unacceptable toxicity or disease progression. The approval was based on phase III clinical trial results first reported at the AACR Annual Meeting 2025 by Ravindra Uppaluri, MD, PhD, of the Brigham and Women’s Hospital, the Dana-Farber Brigham Cancer Center, and Harvard Medical School.

The Oncology Center of Excellence (OCE) Rare Cancers Program at the FDA promotes the development of safe and effective new drugs and biologics to treat patients with rare cancers. Although there is no universally accepted definition for a rare cancer, the Orphan Drug Act defines a rare disease or condition as one which affects less than 200,000 people in the United States. The National Institute of Health defines rare cancers as those that affect fewer than 40,000 people in the U.S., and many cancer types are even rarer.