Notizia - Recent Pharma, Healthcare and Biotech Happenings
Mar 11, 2025
Table of Contents
Neurotech Pharmaceuticals, Inc. has announced that the FDA has approved ENCELTO (revakinagene taroretcel-lwey) for the treatment of Macular Telangiectasia type 2 (MacTel), a progressive neurodegenerative retinal disease that leads to irreversible vision loss. ENCELTO is the first and only FDA-approved treatment for MacTel and utilizes an innovative encapsulated cell therapy technology to deliver continuous therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina, helping to slow disease progression. The approval was based on results from two Phase III clinical trials, which demonstrated that the implant significantly slowed macular photoreceptor loss over 24 months. ENCELTO is expected to become available to patients in the U.S. starting in June 2025.
“This is a historic moment for the MacTel community, as ENCELTO becomes the first-ever FDA-approved treatment for this vision-threatening disease,” said Dr. Thomas M. Aaberg Jr., Chief Medical Officer at Neurotech. “For those affected by MacTel and everyone who has supported this journey, today marks the beginning of a future where vision loss from MacTel may be slowed.” Echoing this sentiment, Dr. Charles C. Wykoff, a clinical investigator from Retinal Consultants of Texas, stated, “I have seen firsthand how MacTel impacts patients’ quality of life. With an FDA-approved treatment now available, I am confident that ENCELTO will help preserve functional vision for many patients over time.”
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Neurotech’s Chief Executive Officer, Richard Small, expressed gratitude to the clinical study participants, investigators, and the entire Neurotech team for making this milestone possible. “Today is an extraordinary day for patients, the retina community, and our company. The approval of ENCELTO represents years of dedicated research and commitment to addressing an unmet need in MacTel treatment,” he said. The breakthrough marks a significant step forward in ophthalmology, offering new hope to those living with this challenging disease.
Plus Therapeutics, Inc. has announced that the FDA has granted Orphan Drug Designation (ODD) to Rhenium (186Re) Obisbemeda for the treatment of leptomeningeal metastases in lung cancer patients. This designation provides key regulatory benefits, including seven years of market exclusivity, tax credits for clinical trials, and exemptions from major regulatory fees, such as the $4.3 million Prescription Drug User Fee Act (PDUFA) charge in 2025. The recognition underscores the growing need for new treatment options for LM, a rare and aggressive complication of lung cancer.
“Receiving Orphan Drug Designation for Rhenium (186Re) Obisbemeda marks a significant milestone in our efforts to develop a much-needed therapy for lung cancer patients with leptomeningeal metastases,” said Mike Rosol, Ph.D., Chief Development Officer at Plus Therapeutics. “These patients currently have limited treatment options, and the growing incidence of LM in lung cancer underscores the urgency for new therapies. This designation, in combination with our previously granted Fast Track designation, strengthens our pathway toward delivering an innovative, targeted radiotherapeutic solution for this highly underserved patient population.”
This milestone follows the recent completion of the ReSPECT-LM Phase I single-dose trial, which established the recommended Phase II dose (RP2D). Plus Therapeutics is now advancing a Phase II single-dose expansion trial and a Phase I multiple-dose trial while actively working with the FDA to outline the optimal pivotal trial strategy. The company remains committed to accelerating the development of Rhenium (186Re) Obisbemeda as a promising targeted therapy for LM patients with lung cancer.
Novo Nordisk has launched NovoCare® Pharmacy, a direct-to-patient delivery service offering all dose strengths of WEGOVY (semaglutide) at a reduced cost of $499 per month for cash-paying patients. This initiative aims to improve access for uninsured individuals or those with commercial insurance that does not cover obesity medications. The launch follows the FDA’s announcement that WEGOVY is no longer in shortage, with all dose strengths now meeting or exceeding projected U.S. demand. NovoCare® Pharmacy is part of Novo Nordisk’s broader efforts to address affordability and expand access to weight management treatments.
“Novo Nordisk continues to advance solutions for patients that improve affordability and access to our medicines, whether they have insurance or not,” said Dave Moore, Executive Vice President of U.S. Operations and Global Business Development. “Today, over 55 million people in the U.S. have coverage for weight management medicines, and 90% of WEGOVY patients with coverage pay $0 to $25 a month. With NovoCare® Pharmacy, patients and prescribers alike have another option for convenient access to all doses of real, FDA-approved WEGOVY at a reduced cost in our high-quality pen.”
NovoCare Pharmacy will provide home delivery of WEGOVY prescriptions through CenterWell Pharmacy, a six-time Specialty Pharmacy Patient Choice award winner. Along with home shipments, the program offers benefit verification, refill reminders, and access to live support from a NovoCare case manager. By ensuring access to authentic, FDA-approved WEGOVY, the service helps patients avoid risks associated with compounded weight-loss drugs.
For those looking to learn more about WEGOVY, we’ve explored this topic in depth in our WEGOVY blog, covering its mechanism, effectiveness, and the latest market trends. With Novo Nordisk’s continued expansion of affordability programs, cash-paying patients using traditional retail pharmacies will soon benefit from similar cost reductions. More details about NovoCare® Pharmacy are available at NovoCare.com.
Capricor Therapeutics announced that the FDA has accepted its Biologics License Application (BLA) for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA granted Priority Review, setting a target action date of August 31, 2025, without identifying any review issues at this time. If approved, deramiocel would become the first therapy specifically for Duchenne-cardiomyopathy, a leading cause of death in DMD patients.
“We are thrilled to announce the acceptance of our BLA, bringing us one step closer to providing this first-in-class treatment for Duchenne-cardiomyopathy, a condition for which there are no approved therapies,” said Linda Marbán, Ph.D., CEO of Capricor. “If our application is successful, we expect deramiocel to be a lifelong treatment, administered quarterly, with the potential for broad adoption across the DMD-cardiomyopathy treatment landscape.”
The BLA submission is based on clinical data from the Phase II HOPE-2 and HOPE-2 Open Label Extension trials, which showed that deramiocel slows disease progression. The FDA has not yet determined whether an Advisory Committee meeting will be required for this application. “Deramiocel has shown in multiple clinical trials attenuation of DMD-cardiomyopathy, which is currently one of the leading causes of death in those with DMD,” said Craig McDonald, M.D., national principal investigator and professor at the University of California, Davis.
The FDA grants Priority Review to therapies that offer significant improvements in treating serious conditions. Deramiocel has received Orphan Drug Designation from both the FDA and EMA and holds RMAT (Regenerative Medicine Advanced Therapy) and ATMP (Advanced Therapy Medicinal Product) designations. If Capricor secures FDA approval for deramiocel by September 30, 2026, the company will be eligible for a Priority Review Voucher (PRV) under the Rare Pediatric Disease Program.
Celltrion has announced that the FDA has approved OMLYCLO (omalizumab-igec) as the first and only biosimilar interchangeable with XOLAIR (omalizumab). This approval expands treatment options for patients with moderate to severe persistent asthma, chronic rhinosinusitis with nasal polyps (CRSwNP), Immunoglobulin E (IgE)-mediated food allergy, and chronic spontaneous urticaria (CSU). The designation as an interchangeable biosimilar ensures that OMLYCLO can be substituted for XOLAIR at the pharmacy level without prescriber intervention, providing more accessible and cost-effective treatment alternatives.
“We are proud to achieve the approval of the first biosimilar to omalizumab in the U.S., which will help broaden access to this important medicine for patients with allergic and respiratory conditions, as well as for physicians, payers, and providers,” said Hetal Patel, Vice President of Medical Affairs at Celltrion USA. “The interchangeability designation of OMLYCLO reinforces confidence among physicians and patients that there is no decrease in effectiveness or increase in safety risk associated with switching between OMLYCLO and the reference product.”
The FDA’s approval was supported by a comprehensive global Phase III clinical trial involving 619 adult patients with CSU, demonstrating comparable efficacy and safety between OMLYCLO and XOLAIR®. Patients were randomized to receive either 300 mg or 150 mg of OMLYCLO or the reference product every four weeks, with follow-ups extending to Week 40. The results confirmed that OMLYCLO maintains equivalent therapeutic benefits and safety profiles, meeting the FDA’s strict criteria for biosimilarity and interchangeability.
“The approval of OMLYCLO could have a meaningful impact for the medical community and patients, offering a high-quality and affordable treatment option while reducing the burden of healthcare costs,” said Thomas Nusbickel, Chief Commercial Officer at Celltrion USA. “With our integrated development, manufacturing, and commercialization platform, Celltrion remains committed to alleviating treatment costs and delivering life-changing medicines to support patients with allergic conditions in the U.S.”
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