Novarits: breakthrough therapy designation to capmatinib for NSCLC, EC approval for Lucentis in preterm infants with ROP

The U.S. Food and Drug Administration granted Breakthrough Therapy Designation to capmatinib (INC280) as a first-line treatment for patients with metastatic MET exon14 skipping-mutated non-small cell lung cancer (NSCLC), said John Tsai, MD, Head of Global Drug Development and Chief Medical Officer, Novartis.

Recent research concludes that the cMET gene is an oncogenic driver, and the investigational lung cancer therapy capmatinib has been shown to be a highly potent and selective MET inhibitor.

The MET mutation is seen in an estimated 3% – 4% of all patients with NSCLC[3]. These patients are generally older and often have a poor prognosis that can limit lung cancer treatment options, Novartis said in a press release.

“As we continue to reimagine medicine and place a renewed focus on the development of innovative lung cancer treatments, we look forward to working with the FDA and global health authorities to bring capmatinib to patients who currently have no available targeted therapy options,” said Dr. Tsai. 

In addition, Novartis separately announced it got EC approval for Lucentis in preterm infants with retinopathy of prematurity (ROP), the first and only licensed pharmacological treatment for these vulnerable infants in the EU.

Approval is based on the landmark RAINBOW study, showing Lucentis (ranibizumab) is an efficacious and safe treatment for infants with ROP, the company said

ROP standard of care treatment, laser surgery, destroys diseased retinal tissue responsible for elevated vascular endothelial growth factor (VEGF) whereas pharmacological therapy, Lucentis, directly targets and reduces VEGF.L

Lucentis (0.2 mg dose) is indicated in preterm infants for the treatment of ROP with zone I (stage 1+, 2+, 3 or 3+), zone II (stage 3+) or AP-ROP (aggressive posterior ROP) disease, making it the only approved pharmacological therapy for this indication in EU.

ROP is a rare eye disease but a leading cause of childhood blindness.

“Everyone deserves the best start in life, so Novartis has reimagined what is possible in the treatment of ROP for this particularly vulnerable patient group,” said Dirk Sauer, Development Unit Head, Novartis Ophthalmology. “The European Commission decision to approve Lucentis in this indication gives clinicians the first and only licensed alternative to laser therapy in the treatment of preterm infants with ROP in the EU. We look forward to providing pediatric retinal specialists with a new therapeutic option for preterm infants with ROP.”