Accelerated Approval Granted for New Treatment for Advanced Soft Tissue Sarcoma
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Accelerated Approval Granted for New Treatment for Advanced Soft Tissue Sarcoma

Treatment works by blocking certain receptors that cause tumor growth

October 24, 2016

The Food and Drug Administration (FDA) has granted accelerated approval to a new treatment for certain types of cancers.

Lartruvo is now approved with chemotherapy medication doxorubicin to treat adults who have certain types of soft tissue sarcoma (STS), cancers that develop in soft tissues such as muscles, fat, and tendons. In combination with doxorubicin, the treatment is approved for patients whose cancer cannot be cured with either radiation or surgery and who also have a type of cancer for which chemotherapy would be appropriate as a treatment.

"For these patients, Lartruvo, added to doxorubicin, provides a new treatment option," said Richard Pazdur, M.D., 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."

Estimations from the National Cancer Institute indicate that 12,310 new STS cases, and almost 5,000 deaths due to the disease, are likely to occur in 2016. In cases of STS that cannot be removed surgically, the most common treatment is either using doxorubicin alone or using it in combination with other medications. Numerous different tumors fall into the category of STS, including those that develop in the muscle, fat, blood vessels, nerves, tendons, or joint linings.

Lartruvo is what is known as a platelet-derived growth factor (PDGF) receptor-alpha blocking antibody. When PDGH receptors are stimulated, they cause tumors to grow. Lartruvo blocks these receptors, helping to slow or stop such tumors from growing.

A randomized clinical trial involving 133 patients with more than 25 different subtypes of metastatic STS was conducted to determine the safety and effectiveness of Lartruvo. Participants received either Lartruvo together with doxorubicin or just doxorubicin by itself. The trial measured how long patients lived after their treatments (overall survival), how long tumors did not grow after the patients received the treatment (progression-free survival), and the percentage of patients whose tumors shrank in response to treatment (overall response rate).

The participants who were treated with both Lartruvo and doxorubicin experienced a statistically significant improvement in their overall survival: the median survival time was 26.5 months in comparison to 14.7 months experienced by patients who were treated with doxorubicin only. Those who received both experienced a median progression-free survival period of 8.2 months, compared with a time of 4.4 months for those with only doxorubicin. And patients who received both treatments experienced a tumor shrinkage rate of 18.2 percent, while those who received only doxorubicin experienced a rate of 7.5 percent.

There are serious risks associated with the use of Lartruvo, including infusion-related reactions, such as low blood pressure, fever, chills, and rash, and embryo-fetal harm. The most common side effects of Lartruvo treatment include nausea, fatigue, low white blood cell levels (neutropenia), musculoskeletal pain, mucous membrane inflammation (mucositis), hair loss (alopecia), vomiting, diarrhea, decreased appetite, abdominal pain, nerve damage (neuropathy), and headache.

The sponsor is now conducting a larger study to further explore Lartruvo's effectiveness in treating the multiple subtypes of STS. Lartruvo also received designation as an orphan drug, which provides incentives like tax credits, user fee waivers, and eligibility for exclusivity to help and encourage development of medications meant to treat rare diseases.