Zevra’s MIPLYFFA Niemann-Pick Disease Approval; FASENRA Approved for Eosinophilic Granulomatosis; Sanofi’s SARCLISA Gets Multiple Myeloma Approval; RYBREVANT Combo Gets FDA Nod for EGFR Lung Cancer; UCB’s BIMZELX Receives Multiple Approval from the FDA

FDA Greenlights Zevra Therapeutics’ MIPLYFFA for Niemann-Pick Disease Type C

Zevra Therapeutics, Inc. has announced that the FDA has approved MIPLYFFA (arimoclomol) capsules as the first oral treatment for Niemann-Pick disease type C. Indicated for use alongside miglustat, MIPLYFFA is intended for both adult and pediatric patients aged two and older. This approval marks a significant milestone for NPC patients, who previously had no FDA-approved options. Zevra has also received a rare pediatric disease priority review voucher in conjunction with this approval.

Neil F. McFarlane, President and CEO of Zevra Therapeutics, emphasized the importance of this approval, stating, “NPC is an ultra-rare, relentlessly progressive, degenerative, and fatal disease for which there were no FDA-approved treatment options until today. The approval of MIPLYFFA is a monumental milestone for NPC patients and their families.” Currently, around 900 individuals in the U.S. are living with NPC, which leads to progressive physical and cognitive limitations.

Laurie Turner, Family Services Manager at the National Niemann-Pick Disease Foundation, expressed gratitude for the long-standing efforts to find treatments: “For more than 30 years, NNPDF and the community have been working to find treatments for NPC, and we are grateful for the diligence and commitment of the researchers, clinicians, families, and Zevra for making this approval possible.”

The FDA approval is based on a 12-month multicenter, randomized, double-blind, placebo-controlled trial that demonstrated MIPLYFFA’s effectiveness when combined with miglustat. The treatment halted disease progression, showing a decrease of 0.2 points on the NPC Clinical Severity Scale compared to 1.9 points of progression for those receiving miglustat alone. Zevra plans to launch MIPLYFFA commercially in the U.S. within eight to twelve weeks, allowing more patients access to this crucial treatment option.

FASENRA Wins FDA Approval for Eosinophilic Granulomatosis with Polyangiitis

AstraZeneca’s FASENRA (benralizumab) has received FDA approval for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis, a rare and potentially fatal immune-mediated vasculitis that can cause organ damage. The approval follows positive outcomes from the MANDARA Phase III trial, published in The New England Journal of Medicine, which compared FASENRA’s efficacy and safety to the only existing EGPA treatment, mepolizumab.

In the MANDARA trial, nearly 60% of patients treated with FASENRA achieved remission, comparable to those treated with mepolizumab. Notably, 41% of FASENRA recipients were able to fully taper off oral corticosteroids, compared to just 26% in the mepolizumab group. This trial marked the first head-to-head non-inferiority study of biologics in EGPA patients, offering promising results for a condition that has limited treatment options.

Dr. Michael Wechsler, an investigator in the MANDARA trial, remarked, “This approval is great news for patients with EGPA in the US who continue to suffer from debilitating symptoms. Benralizumab is a much-needed treatment option, with data showing that not only is remission an achievable goal for EGPA patients, but benralizumab can also help patients taper off steroid therapy.”

Joyce Kullman, Executive Director of the Vasculitis Foundation, noted the importance of this approval for the approximately 15,000 patients in the U.S. living with EGPA, stating, “The approval of another treatment in EGPA is welcome news. Patients need more treatment options.” With this new approval, FASENRA provides physicians in the U.S. with a convenient single monthly subcutaneous injection option for their EGPA patients, expanding the potential of FASENRA beyond severe eosinophilic asthma.

SARCLISA Becomes First Anti-CD38 Therapy for Newly Diagnosed Multiple Myeloma Patients

Sanofi’s SARCLISA (isatuximab) has been approved by the FDA as the first anti-CD38 therapy in combination with standard-of-care treatment for adult patients with newly diagnosed multiple myeloma who are not eligible for autologous stem cell transplant. This approval is based on positive results from the IMROZ Phase III study, which demonstrated that SARCLISA, when combined with bortezomib, lenalidomide, and dexamethasone, significantly improved progression-free survival (PFS) by 40% compared to VRd alone.

Dr. Thomas Martin, Clinical Professor of Medicine at the University of California San Francisco, highlighted the importance of this approval: “Multiple myeloma is most frequently diagnosed in patients 65 years and older… The significant clinical benefit and improvements in progression-free survival… make today’s approval an important moment for this vulnerable patient population and the larger multiple myeloma community.” This marks the third indication for SARCLISA in the U.S. and the first for newly diagnosed patients.

Brian Foard, Executive Vice President at Sanofi, stated, “The FDA’s decision marks another momentous milestoneWith today’s approval, doctors now have an important new option at their disposal that’s been shown to slow disease progression for longer compared to the current standard-of-care.” The IMROZ study, which formed the basis of the FDA approval, found that SARCLISA combined with VRd led to a median PFS that was not reached, compared to 54.3 months for VRd alone.

The safety profile of SARCLISA in the study was consistent with its known profile, with common adverse reactions including upper respiratory tract infections, diarrhea, and fatigue. Serious adverse reactions were reported in 71% of patients receiving the combination therapy, with pneumonia being the most frequent. SARCLISA is now approved in over 50 countries for treating relapsed or refractory disease, further emphasizing Sanofi’s commitment to addressing the critical care gap in multiple myeloma.

FDA Approves RYBREVANT Combo as First Targeted Therapy for EGFR-Mutated Lung Cancer, Cutting Progression Risk

Johnson & Johnson announced that the FDA has approved RYBREVANT (amivantamab-vmjw) in combination with standard chemotherapy (carboplatin and pemetrexed) for adult patients with locally advanced or metastatic non-small cell lung cancer featuring epidermal growth factor receptor exon 19 deletions or L858R substitution mutations, following disease progression on an EGFR tyrosine kinase inhibitor.

Dr. Martin Dietrich, an oncologist at the Cancer Care Centers of Brevard, highlighted the significance of this approval, stating, “RYBREVANT plus chemotherapy may address the most common mechanisms of treatment resistance.offering an important new second-line option for patients.” With a five-year survival rate of less than 20% for advanced EGFR-mutated NSCLC, effective treatment options are crucial.

The FDA’s decision is based on the Phase III MARIPOSA-2 study, which showed that RYBREVANT plus chemotherapy reduced the risk of disease progression or death (progression-free survival [PFS]) by 52% compared to chemotherapy alone. The combination’s median PFS was 6.3 months versus 4.2 months for chemotherapy alone. The overall response rate (ORR) was 53% for RYBREVANT® compared to 29% for chemotherapy.

Andrea Ferris, President and CEO of LUNGevity Foundation, expressed optimism: “New therapeutic options like RYBREVANT and chemotherapy help address unmet needs.” RYBREVANT is now the only NCCN® Category 1 treatment option for symptomatic patients with EGFR-mutated NSCLC progressing on osimertinib. The safety profile was consistent with established treatments, with permanent discontinuation due to adverse reactions occurring in 11% of patients. This approval marks a significant advancement for the nearly 30,000 patients diagnosed with EGFR-mutated NSCLC in the U.S. each year.

UCB’s BIMZELX Receives FDA Nod for Psoriatic Arthritis, Axial Spondyloarthritis, and Ankylosing Spondylitis

UCB announced that the FDA has approved BIMZELX (bimekizumab-bkzx) for adults with active psoriatic arthritis, active non-radiographic axial spondyloarthritis with objective signs of inflammation, and active ankylosing spondylitis. This makes BIMZELX the first and only IL-17A and IL-17F inhibitor approved in the U.S. for these four chronic immune-mediated inflammatory diseases.

Two Phase III studies support the approval for active PsA by demonstrating significant improvements in joint and skin symptoms at Week 16 compared to placebo, with benefits sustained to Week 52. Data showing statistically significant improvements in signs and symptoms at Week 16, also sustained to Week 52, supported the approvals for active nr-axSpA and ankylosing spondylitis.

Emmanuel Caeymaex, UCB’s Executive Vice President, emphasized the importance of this dual inhibition, stating it provides meaningful outcomes for patients. The recommended dosage for adults with these conditions is 160 mg via subcutaneous injection every four weeks.

Phase III studies, BE OPTIMAL and BE COMPLETE, demonstrated that bimekizumab-bkzx achieved clinically significant responses in both biologic-naïve and TNF inhibitor-inadequate responder populations. Leah M. Howard, President and CEO of the National Psoriasis Foundation, noted the approval brings hope for better management of PsA symptoms.

The BE MOBILE 1 and BE MOBILE 2 studies supported the approvals for nr-axSpA and AS, showing sustained clinical responses at Week 52. Seth Ginsberg, Co-Founder of the Global Health Living Foundation, highlighted the positive impact of new treatment options for patients facing daily challenges due to their conditions.