Rigel Pharmaceuticals Finalizes GAVRETO® (pralsetinib) NDA Transfer

Rigel Pharmaceuticals, Inc. has officially completed the transfer of the New Drug Application for GAVRETO® (pralsetinib) to its portfolio. This drug targets adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) and adult and pediatric patients 12 years and older with advanced RET fusion-positive thyroid cancer requiring systemic therapy. GAVRETO will be available in the U.S. starting June 27, 2024, through Rigel's distribution channels.

Raul Rodriguez, Rigel's president and CEO, expressed enthusiasm about the completion of the transfer process and the imminent availability of GAVRETO. He emphasized the company's readiness to support both existing and new patients, ensuring seamless access to this critical treatment. Rodriguez highlighted that incorporating GAVRETO into Rigel's commercial offerings aligns with the company's strategy to enhance its presence in hematology and oncology sectors.

The U.S. Food and Drug Administration (FDA) has fully approved GAVRETO for NSCLC, while its use for advanced thyroid cancer is granted under accelerated approval, contingent on further clinical trials confirming its benefits. Rigel is currently in discussions with the FDA about these confirmatory requirements to ensure continued approval for thyroid cancer indications.

GAVRETO stands out as the only once-daily, oral RET-inhibitor therapy, specifically designed for patients with RET fusion-positive mNSCLC and advanced thyroid carcinoma. The recommended dosage is 400 mg taken orally once a day. Rigel will offer patient assistance programs to help navigate insurance coverage, provide financial aid, and support through their RIGEL ONECARE® service center.

In February 2024, Rigel announced its acquisition of the U.S. commercial rights for GAVRETO from Blueprint Medicines Corporation, marking a significant expansion in its commercial portfolio.

Lung cancer remains the leading cause of cancer-related deaths in the U.S., with over 230,000 new cases expected in 2024. NSCLC accounts for the majority of these cases, and RET fusions are found in approximately 1-2% of NSCLC patients. GAVRETO aims to address this specific patient group.

GAVRETO's indications cover a broad spectrum of patients. It is prescribed for adults with metastatic RET fusion-positive NSCLC and for adult and pediatric patients aged 12 years and older with advanced RET fusion-positive thyroid cancer who are not suitable candidates for radioactive iodine therapy. The accelerated approval for thyroid cancer is based on the overall response rate and the duration of response, with future clinical trials required to verify these benefits.

Safety information for GAVRETO indicates potential risks such as Interstitial Lung Disease (ILD)/Pneumonitis, hypertension, hepatotoxicity, hemorrhagic events, and Tumor Lysis Syndrome (TLS). Patients should be closely monitored for these conditions, and the drug should be withheld or discontinued based on the severity of adverse reactions. Women who are pregnant or breastfeeding are advised to avoid GAVRETO due to potential harm to the fetus and the unknown effects on infant health during lactation.

The drug's common side effects include musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, fever, and cough. Laboratory abnormalities observed in clinical trials include changes in blood counts, liver enzymes, and electrolytes.

Patients are advised to avoid coadministration of GAVRETO with CYP3A inhibitors, P-gp inhibitors, and inducers, which can affect the drug's metabolism. Proper blood pressure management and monitoring of liver function are crucial during GAVRETO therapy.

In conclusion, Rigel Pharmaceuticals is prepared to launch GAVRETO, providing an important treatment option for patients with specific types of lung and thyroid cancer. The company's infrastructure and support programs aim to ensure patients receive uninterrupted access to this new therapeutic option.