FDA Grants Accelerated Approval to Olaratumab for Treatment of Advanced Soft-Tissue Sarcoma

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This is the first new therapy approved by the FDA for the initial treatment of soft-tissue sarcoma since doxorubicin's approval more than 40 years ago.
— Richard Pazdur, MD

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Today, the U.S. Food and Drug Administration (FDA) granted accelerated approval to olaratumab (Lartruvo) in combination with doxorubicin to treat adults with certain types of soft-tissue sarcoma. Olaratumab is approved for use with the FDA-approved chemotherapy drug doxorubicin for the treatment of patients with soft-tissue sarcoma who cannot be cured with radiation or surgery and who have a type of sarcoma for which an anthracycline is an appropriate treatment.

New Treatment Option

“For these patients, [olaratumab], added to doxorubicin, provides a new treatment option,” said Richard Pazdur, MD, Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and Acting Director of the FDA’s Oncology Center of Excellence. “This is the first new therapy approved by the FDA for the initial treatment of soft-tissue sarcoma since doxorubicin’s approval more than 40 years ago.”

The National Cancer Institute estimates that 12,310 new cases of soft-tissue sarcoma and nearly 5,000 deaths are likely to occur from the disease in 2016. The most common treatment for soft-tissue sarcoma that cannot be removed by surgery is treatment with doxorubicin alone or with other drugs.

Olaratumab is a platelet-derived growth factor (PDGF) receptor-alpha blocking antibody. Olaratumab works by blocking PDGF receptors, which may help slow or stop tumor growth.

Trial Results

The safety and efficacy of olaratumab were studied in a randomized clinical trial involving 133 patients with more than 25 different subtypes of metastatic soft-tissue sarcoma. Patients received either olaratumab with doxorubicin or doxorubicin alone. This trial measured overall survival, progression-free survival, and the overall response rate.

Patients in this trial who received olaratumab with doxorubicin had a statistically significant improvement in overall survival: Their median survival was 26.5 months compared to 14.7 months for patients who received doxorubicin alone. Patients who received olaratumab with doxorubicin had a median progression-free survival of 8.2 months compared to 4.4 months for patients who received doxorubicin alone. Tumor shrinkage was 18.2% for patients who received olaratumab with doxorubicin and 7.5% for those who received doxorubicin alone.

Olaratumab is associated with serious risks, including infusion-related reactions and embryofetal harm. Infusion-related reactions include low blood pressure, fever, chills, and rash. The most common side effects of treatment with olaratumab are nausea, fatigue, neutropenia, musculoskeletal pain, mucositis, alopecia, vomiting, diarrhea, decreased appetite, abdominal pain, neuropathy, and headache.

Accelerated Approval

The FDA is approving olaratumab under the agency’s Accelerated Approval program, which allows approval of a drug to treat a serious or life-threatening disease or condition based on clinical data showing the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit. The sponsor, Eli Lilly and Company, is conducting a larger study, which is currently underway, to further explore the effectiveness of olaratumab across the multiple subtypes of soft-tissue sarcoma.

For more details about the use of olaratumab, see In the Clinic here. ■

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