In addition to the European approval, WINREVAIR was also granted FDA approval in the U.S. on March 26, 2024. Dr. Marc Humbert of Université Paris-Saclay emphasized the impact of the treatment, stating, “WINREVAIR should be considered as a new standard of care for treating PAH patients.” WINREVAIR is administered every three weeks via a single injection and can be given by patients or caregivers with proper training.
European Commission Approves BALVERSA for Adult Patients With Unresectable or Metastatic Urothelial Carcinoma
Janssen-Cilag International NV, a Johnson & Johnson company, announced that the European Commission (EC) has approved BALVERSA® (erdafitinib) as a once-daily oral monotherapy for adult patients with unresectable or metastatic urothelial carcinoma (mUC) harboring FGFR3 genetic alterations. This approval targets patients who have previously undergone at least one line of therapy with a PD-1 or PD-L1 inhibitor.
Dr. Yohann Loriot from Institut Gustave Roussy commented, “Bladder cancer is one of Europe’s most common cancers, and the need for innovative treatment options remains high. Erdafitinib is a novel, targeted therapy that has been shown to significantly improve overall and progression-free survival for patients with FGFR3 alterations.”
The approval is based on the Phase III THOR study results, which demonstrated that erdafitinib significantly improved median overall survival (OS) to 12.1 months compared to 7.8 months with chemotherapy. Additionally, median progression-free survival (PFS) was 5.6 months with erdafitinib versus 2.7 months with chemotherapy. Serious treatment-related adverse events were lower with erdafitinib, showing a better overall response rate than chemotherapy.
Henar Hevia from Johnson & Johnson emphasized, “The approval of erdafitinib as a precision therapy further highlights the importance of FGFR testing for all patients with metastatic urothelial cancer.” Kiran Patel, Vice President at Johnson & Johnson, added, “The EC approval of erdafitinib reflects our unwavering commitment to transforming outcomes for people living with unresectable or metastatic urothelial carcinoma.”
Novartis, Versant Launch RNA Kidney Medicines—Focused Borealis
Novartis and Versant Ventures have announced the launch of Borealis Biosciences, a new biotech company focused on developing RNA therapeutics for kidney diseases, with $150 million in Series A financing. This collaboration builds on their previous success with Chinook Therapeutics, which Versant founded in 2019 and was acquired by Novartis in 2023 for $3.2 billion, plus an additional $300 million in potential milestones. The new venture aims to address unmet needs in kidney disease treatment by leveraging RNA-based technologies.
Borealis Biosciences will utilize Novartis’ xRNA platform, which modulates disease-causing proteins through natural mRNA. Despite progress with drugs like atrasentan, which is under FDA review for immunoglobulin A nephropathy (IgAN), traditional therapies have struggled with certain targets.
Jerel Davis from Versant, who will oversee operations at Borealis, noted, “We’ve recognized over the last six years that some of the most validated targets for kidney disease have been out of reach with traditional modalities.”
The company will operate out of a 23,000-square-foot facility in Vancouver previously used by Chinook. Novartis has committed up to $100 million upfront, with additional funding for research, and will have the option to acquire up to two Borealis programs for up to $750 million in total. Ronny Gal from Novartis highlighted the deal as a testament to the company’s commitment to advancing renal science, stating, “A first of its kind for Novartis, this three-part transaction of divestment, collaboration and investment is a testament to our company’s unwavering focus on advancing renal science.”
Moderna Receives European Commission Approval for RSV Vaccine mRESVIA(R)
Moderna, Inc. has received marketing authorization from the European Commission (EC) for mRESVIA® (mRNA-1345), an mRNA vaccine designed to protect adults aged 60 and older from lower respiratory tract disease caused by respiratory syncytial virus (RSV). This authorization follows a Positive Opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) and is valid across all 27 EU member states, as well as Iceland, Liechtenstein, and Norway.
Stéphane Bancel, Moderna’s CEO, stated, “The European Commission’s approval of mRESVIA is an important milestone for public health and highlights Moderna’s mRNA leadership.”
RSV is a highly contagious virus leading to significant disease burden in both infants and older adults, with an estimated 160,000 hospital admissions annually in the EU, predominantly among those aged 65 and over. The marketing authorization is supported by data from the Phase III ConquerRSV trial, involving around 37,000 adults. The trial showed an 83.7% vaccine efficacy against RSV lower respiratory tract disease during the initial follow-up period and sustained efficacy of 63.3% in extended follow-up. The most common side effects were injection site pain, fatigue, headache, myalgia, and arthralgia.
In addition to European approval, mRESVIA was also approved by the FDA in May 2024 under a breakthrough therapy designation, marking Moderna’s second approved mRNA product. Moderna continues to seek marketing authorization for mRNA-1345 in various global markets.
FDA Approves RYBREVANT® and LAZCLUZE™ as a First-line, Chemotherapy-free Treatment for EGFR-mutated Advanced Lung Cancer
Johnson & Johnson announced that the FDA has approved RYBREVANT® (amivantamab-vmjw) in combination with LAZCLUZE™ (lazertinib) for the first-line treatment of adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 19 deletions or exon 21 L858R mutations. This approval marks the first multitargeted, chemotherapy-free regimen demonstrated to be superior to osimertinib for treating EGFR-mutated NSCLC.
The approval is based on positive results from the Phase III MARIPOSA study, which showed that RYBREVANT® plus LAZCLUZE™ reduced the risk of disease progression or death by 30% compared to osimertinib, with a median progression-free survival of 23.7 months versus 16.6 months. The median duration of response was also significantly longer with the combination therapy.
Dr. Alexander Spira, a study investigator, highlighted the therapy’s potential to set a new standard of care, stating, “Patients will now have the option of a potential new first-line standard of care with significant clinical benefits over osimertinib.”
Jennifer Taubert, Executive Vice President at Johnson & Johnson, emphasized the significance of this approval, noting, “RYBREVANT plus LAZCLUZE establishes a new benchmark in the advanced first-line setting.” John Reed, EVP of Innovative Medicine R&D, added, “Today’s FDA approval of chemotherapy-free RYBREVANT plus LAZCLUZE in the first line is an incredible step towards our goal of altering the trajectory of lung cancer.” The combination’s safety profile was consistent with that of the individual treatments, with venous thromboembolic events observed.
