The long weekend saw BMS’s Opdivo, Novartis’s Kymriah and Roche’s Evrysdi win FDA approvals in new indications.
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The long weekend saw three established drugs developed by Bristol Myers Squibb, Novartis and Roche score new approvals from the U.S. Food and Drug Administration.
Opdivo Now a First-Line Therapy for Esophageal Squamous Cell Carcinoma
Bristol Myers Squibb’s Opdivo (nivolumab) has been greenlit for use alongside Yervoy (ipilimumab) as a first-line therapy for adults diagnosed with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC). Patients are to be given the combination intravenously regardless of PD-L1 status.
The FDA’s decision is based on results from the Phase III CheckMate -648 trial, the largest Phase III study of immunotherapy in first-line ESCC, which assessed Opdivo with chemotherapy and Opdivo plus Yervoy versus chemotherapy alone.
The CheckMate -648 trial demonstrated Opdivo plus chemotherapy to be superior to administering chemotherapy alone in all randomized patients, logging a median overall survival rate of 13.2 months versus 10.7 months. In all patients, the median progression-free survival rate was 5.8 months for Opdivo with chemotherapy compared to 5.6 months in just chemo.
Meanwhile, Opdivo plus Yervoy also demonstrated improved median overall survival (mOS) at 12.8 months versus 10.7 months in those who received only chemotherapy in all randomized patients. In those whose tumors express PD-L1, the mOS with Opdivo plus Yervoy was 13.7 months versus 9.1 months in chemo alone.
“The approvals bring two first-line immunotherapy-based treatment options at once, Opdivo in combination with chemotherapy and Opdivo plus Yervoy as the first dual immunotherapy option, to newly diagnosed patients with unresectable advanced or metastatic esophageal squamous cell carcinoma, further building on the role of Opdivo-based regimens in upper gastroesophageal cancers,” Adam Lenkowsky, SVP and general manager for the U.S. cardiovascular, immunology and oncology unit at BMS, said in a statement.
Novartis’s Kymriah Scores Third Approval
The second FDA approval is for Novartis’s Kymriah for adults with relapsed or refractory follicular lymphoma who have received two or more lines of systemic therapy. This represents the third FDA approval for Kymriah (tisagenlecleucel), which is also the only CAR-T cell therapy that is allowed for use in both pediatric and adult patients.
The latest FDA decision is based on positive results from the Phase II ELARA study, which evaluated 90 patients followed closely for around 17 months. About 86% of the trial participants demonstrated a response, including 68% who reported a complete response. Kymriah also produced a prolonged durable response in 85% of participants, with a complete response 12 months after the initial one. The drug is said to be effective in high-risk patients, even those diagnosed with POD24, refractory disease, bulky disease or those with high Follicular Lymphoma International Prognostic Index (FLIPI) scores.
“The approval of Kymriah offers patients with relapsed or refractory follicular lymphoma a new treatment option and new hope for improving patient outcomes. Having this single infusion treatment option helps to transform the way healthcare providers approach this type of blood cancer and we commend those who have contributed to the acceleration of scientific research for the benefit of patients,” Meghan Gutierrez, chief executive officer of the Lymphoma Research Foundation noted in a press release.
The U.S. is not the first to approve Kymriah for adults with r/r FL after two or more lines of systemic therapy. In May 2022, the European Commission allowed the drug for this third indication in the EU.
Roche SMA Drug Approved for Pre-symptomatic Infants
Wrapping up the trio of FDA approvals is Roche’s Evrysdi (risdiplam)'s label extension to include babies under two months old who are diagnosed with spinal muscular atrophy.
The extended indication follows positive results from the RAINBOWFISH trial in newborns, which demonstrated the drug’s ability to hit key milestones in pre-symptomatic babies. Such milestones included sitting and standing with half walking 12 months after the start of treatment. As of this writing, Evrysdi has the FDA’s go signal to treat SMA in all ages.
In addition, the drug’s prescribing information was updated to cover the latest two-year pooled data from FIREFISH Part 1 and Part 2. The two trials demonstrated the long-term effectiveness and safety in symptomatic patients living with Type 1 SMA.
“The priority review and subsequent approval of Evrysdi for babies under two months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” Levi Garraway, M.D., Ph. D., chief medical officer and head of global product development at Roche said. “We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers.”
Evrysdi is now approved in 81 countries, and its dossier is being reviewed in another 27 countries. Roche leads the development activities for the drug alongside PTC Therapeutics and the SMA Foundation.
