Tuesday, June 18, 2024

Pediatric Cancers: Lilly’s Retevmo Approved for Treating RET-Positive Pediatric Cancers

Similar articles

Pediatric cancers have led the US Food and Drug Administration (FDA) to grant accelerated approval for Eli Lilly’s RET inhibitor, Retevmo (selpercatinib), as a treatment for various RET-altered cancers in children under 12 years of age.

The newly approved indication covers advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, advanced or metastatic thyroid cancer with a RET gene fusion that cannot be treated with radioactive iodine therapy, and locally advanced or metastatic solid tumors with a RET gene fusion that have progressed after prior systemic treatment or have no other treatment options.

Previously, the FDA had approved Retevmo for treating RET-positive thyroid cancer in adults and children aged 12 years and older, as well as RET-positive solid tumors in adults only. Lilly acquired Retevmo through its $8 billion acquisition of Loxo Oncology in 2019.

The new FDA approval is based on the results of the single-arm LIBRETTO-121 study, which included 25 patients aged two to 20 years. The study showed an overall response rate of 48%, with a median duration of response not reached after 12 months of follow-up. At that time, 92% of the responding subjects remained in response. The most common adverse reactions observed in more than 25% of patients included musculoskeletal and abdominal pain, diarrhea, headache, nausea, vomiting, fatigue, and bleeding, among others.

This new approval is expected to enhance Retevmo’s position in the market, providing a significant advantage over its main competitor, Blueprint Medicines’ Gavreto (pralsetinib). Currently, Gavreto is approved by the FDA for RET-positive thyroid cancer and non-small cell lung cancer (NSCLC). The broader approval for Retevmo could lead to increased usage and better sales performance.

pediatric cancers

RET-Positive Pediatric Cancers: Diverse Tumor Types and Market Growth

RET-positive cancers predominantly include lung and thyroid tumors, but RET mutations are also found in pancreatic, colorectal, breast, and salivary gland cancers. Retevmo generated sales of $254 million last year, showing a growth of one-third compared to the previous year. This performance is well ahead of Gavreto, which reported sales of $60 million last year, including $28 million from the US market. Gavreto was previously partnered with Roche until the alliance ended in February. Blueprint Medicines has since partnered with Rigel Pharma for the US market, while the development and marketing of Gavreto in other regions are winding down, except in China where it is licensed to Innovent Biologics and was approved in 2022.

The new approval for Retevmo is significant as it addresses a critical need for effective treatment options for children with RET-altered cancers. Dr. Luigi Manenti, Chief Medical Officer of Alentis, highlighted the urgent need for effective IPF treatments, emphasizing the potential of targeting CLDN1 in fibrotic lungs with highly specific antibodies. Dr. Steven Nathan, Medical Director of the Advanced Lung Disease and Transplant Program at Inova Fairfax Hospital, praised the development of lixudebart for its favorable safety profile in Phase 1 trials and its potential to address the root causes of IPF.

Orphan Drug designation by the FDA provides incentives such as tax credits for clinical trials, user-fee exemptions, and potential market exclusivity for seven years post-approval. Lixudebart is a first-in-class monoclonal antibody designed to reverse organ fibrosis by targeting a unique CLDN1 epitope in fibrotic tissue. It has shown promising results in Phase 1 studies and is currently being tested in clinical trials for advanced liver fibrosis and ANCA-associated vasculitis.

You can follow our news on our Telegram and LinkedIn accounts.

Idiopathic Pulmonary Fibrosis (IPF) is a rare, serious chronic disease affecting lung tissue, leading to fibrosis and making breathing increasingly difficult. There is no cure for IPF, and existing treatments only slow disease progression. Alentis Therapeutics is committed to developing innovative treatments targeting the root causes of fibrotic diseases, with lixudebart showing potential as a transformative therapy for IPF.

Alentis, known as the CLDN1 company, is a clinical-stage biotech developing treatments for CLDN1+ tumors and organ fibrosis. CLDN1 plays a key role in cancer and fibrotic disease pathology. Alentis is pioneering anti-CLDN1 antibodies and antibody-drug conjugates (ADCs) to modify and reverse the course of these diseases, aiming to provide new treatment options for patients with significant unmet medical needs.

Subscribe to our newsletter

To be updated with all the latest news, offers and special announcements.

Latest article