FDA Approves Verastem’s Ovarian Cancer Therapy for Some Adult Patients

It marks the first and only approval for a drug to treat adults with low-grade serous ovarian cancer.

The U.S. Food and Drug Administration building in White Oak, Md., on June 5, 2023. Madalina Vasiliu/The Epoch Times

The Food and Drug Administration (FDA) has granted accelerated approval to biopharmaceutical company Verastem Oncology’s combination therapy for adults who have received prior treatment for a rare type of ovarian cancer, the regulator announced on Thursday.

The FDA approved Avmapki Fakzynja co-pack (avutometinib capsules; defactinib tablets) for patients with recurrent Kirsten rat sarcoma viral oncogene homolog (KRAS) mutated low-grade serous ovarian cancer (LGSOC) who received prior systemic therapy.

It marks the first and only approval for a drug to treat adults with this form of cancer, the Massachusetts-based drug maker said.

The new therapy will be available in the United States in one week at a list price of $48,500 for a 28-day prescription.

LGSOC is a rare ovarian cancer that is both insidious and persistent, according to Verastem. It is distinct and dissimilar from high-grade serous ovarian cancer (HGSOC) in that it is highly recurrent and less sensitive to chemotherapy, meaning it requires different treatment, such as hormone therapy.

Approximately 6,000–8,000 women in the United States and 80,000 worldwide are living with this disease, which is typically diagnosed in those in the age ranges of 20–30 and 50–60 and has a median survival of approximately 10 years, according to Verastem.

Dan Paterson, president and chief executive officer of Verastem Oncology, said the approval represents “not only the first-ever FDA-approved treatment specifically for this rare cancer but also a new day for people living with this disease who have been in desperate need of new treatment options.”

“We are very proud to bring two innovative medicines in one combination treatment to the LGSOC community,” Paterson said. “We thank the researchers, patients, and their families participating in our clinical trials, the patient advocacy community, the FDA, and everyone at Verastem for their dedication and commitment to helping us bring AVMAPKI FAKZYNJA CO-PACK to patients in the U.S.”

Approval of Avmapki Fakzynja was based on data from a mid-stage trial of 57 patients with KRAS-mutated recurrent LGSOC who were treated with at least one prior therapy, including a platinum-based regimen, the FDA said.

Patients received 3.2 mg of Avmapki orally twice weekly for three weeks and 200 mg of Fakzynja orally twice daily for three weeks until disease progression or unacceptable toxicity.

In the trial, Verastem’s drug demonstrated a 44 percent overall response rate and was generally well tolerated, the FDA stated.

The most common adverse reactions included nausea, fatigue, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, vomiting, abdominal pain, decreased platelet count, constipation, dry skin, and urinary tract infection, among others, according to the FDA.

Verastem said possible serious side effects also include ocular disorders, skin toxicities, hepatotoxicity, rhabdomyolysis, and fetal harm when administered during pregnancy.

The combination drug works by inhibiting multiple pathways that promote cancer cell survival and growth and drug resistance.

Shares of Verastem Oncology climbed 5 percent in afternoon trading and are currently trading at $6.94.