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India Advances Rare Disease Treatment Pipeline with Nine New Medications

Drug Approval
  • India has successfully launched five medications for seven rare diseases including Thalassemia, Wilson's disease, and Cystic Fibrosis, with four additional drugs expected to reach market soon.
  • The National Policy for Rare Diseases currently covers 63 rare conditions, with NITI Aayog prioritizing 13 disorders for accelerated development in 2023.
  • Generic medications from local companies are providing significant cost savings for rare disease patients, while the government's PLI scheme supports production of treatments for eight rare diseases.
  • With 70-90 million Indians affected by rare diseases out of 300 million globally, industry leaders emphasize the need for improved R&D ecosystems and faster regulatory processes.

Natco Pharma Launches Generic Bosentan for Pediatric Pulmonary Arterial Hypertension with 180-Day US Market Exclusivity

Drug Approval
  • Natco Pharma has launched generic Bosentan tablets (32 mg) for oral suspension in the US, securing 180-day market exclusivity as the first-to-file generic manufacturer.
  • The drug treats pulmonary arterial hypertension in pediatric patients aged 3 years and older, serving as a generic equivalent to Actelion's Tracleer tablets.
  • Lupin Ltd serves as the marketing partner for this product, which had estimated annual sales of USD 10 million in the US market according to recent industry data.
  • The launch represents a significant opportunity in the specialized pediatric PAH treatment market, addressing an important unmet need for affordable therapy options.

FDA Approves First Continuous Glucose Monitor for Weight Management Beyond Diabetes

Drug Approval
  • The FDA has approved Signos's continuous glucose monitoring system as the first-ever CGM specifically designed for weight management, extending access beyond traditional diabetes care to any consumer seeking weight loss support.
  • The system combines an AI platform with Dexcom's off-the-shelf CGM technology to provide real-time glucose data and personalized lifestyle recommendations, costing $129-139 for multi-month plans compared to roughly $1,000 monthly for GLP-1 medications.
  • With nearly 74% of Americans classified as overweight or obese and obesity costing the U.S. healthcare system over $170 billion annually, this approval represents a significant shift toward accessible, personalized weight management technology.
  • The device can be used independently or in combination with existing treatments like GLP-1 drugs or bariatric surgery, offering support for individuals across the weight loss spectrum from five to 100 pounds.

Health Canada Approves Iovance's AMTAGVI as First T-Cell Therapy for Solid Tumors in Canada

Drug Approval
  • Health Canada granted conditional marketing authorization for AMTAGVI, marking the first T-cell therapy approved for solid tumors in Canada, specifically for advanced melanoma treatment.
  • The approval is based on safety and efficacy data from the mid-stage C-144-01 trial, with conditional status pending confirmatory trials to demonstrate clinical benefit.
  • AMTAGVI is indicated for adult patients with unresectable or metastatic melanoma who have progressed after at least one prior systemic therapy and lack satisfactory treatment alternatives.
  • Iovance plans to authorize its first Canadian treatment center within months and continues pursuing approvals in additional international markets including the U.K., Australia, and Switzerland.

Health Canada Approves Ozempic for Kidney Protection in Type 2 Diabetes Patients

Drug Approval
  • Health Canada approved Ozempic on August 13 to reduce kidney deterioration risk in Type 2 diabetes patients with chronic kidney disease.
  • The international FLOW trial demonstrated a 24% reduction in kidney failure risk among 3,533 patients compared to placebo over 3.4 years.
  • Ozempic becomes the first GLP-1 drug approved in Canada to protect both kidneys and heart in diabetic patients.
  • Between 30-50% of Type 2 diabetes patients develop chronic kidney disease, making this approval clinically significant.

Xspray Pharma Submits Improved Nilotinib Formulation XS003 to FDA for Chronic Myeloid Leukemia Treatment

Drug Approval
  • Xspray Pharma has submitted a New Drug Application to the FDA for XS003, an improved nilotinib formulation for chronic myeloid leukemia treatment using proprietary HyNap technology.
  • XS003 demonstrates the lowest documented food interaction within the nilotinib class and improved dose linearity, potentially eliminating the three-hour fasting requirement of the reference product Tasigna.
  • Registration studies show bioequivalence with Tasigna despite XS003 being administered at less than half the dose, with only 28% food interaction compared to 82% for the reference product.
  • The FDA is expected to initiate review within 60 days, with a regulatory decision anticipated approximately eight months thereafter, targeting a U.S. nilotinib market worth $850 million.

Roche Canada Secures Public Funding for Vabysmo in Quebec for Retinal Vein Occlusion Treatment

Drug Approval
  • Hoffmann-La Roche Limited announced that Vabysmo (faricimab injection) is now publicly funded for macular edema secondary to retinal vein occlusion on Quebec's RAMQ medication list.
  • The funding includes coverage for Vabysmo in a 6.0 mg single-use pre-filled syringe format, expanding treatment options for Quebec patients.
  • Vabysmo is now covered in Quebec for all three of its approved indications including neovascular age-related macular degeneration, diabetic macular edema, and macular edema secondary to RVO.
  • The bispecific antibody works by inhibiting both Ang-2 and VEGF-A pathways and has received authorization in over 100 countries for various retinal conditions.

Over-the-Counter Birth Control Pill Dramatically Increases Contraceptive Access Among Underserved Populations

Drug Approval
  • A nationwide OHSU study of 986 people across 49 states found a 31.8 percentage point increase in contraceptive use among those who previously used no birth control method after accessing over-the-counter pills.
  • The over-the-counter birth control pill particularly benefited racial and ethnic minorities, adolescents, uninsured individuals, and Medicaid recipients who faced the greatest barriers to reproductive healthcare access.
  • Research published in JAMA Network Open demonstrates that removing prescription requirements for oral contraceptives effectively expands access for underserved populations, especially in rural areas where healthcare access is limited.
  • The study provides clear evidence that the FDA's 2023 approval of over-the-counter birth control pills is addressing critical gaps in contraceptive access during a time of increasing reproductive health restrictions.

Sino Biopharm Reports Strong H1 2025 Results with 27% Growth in Innovative Drug Sales and Strategic LaNova Acquisition

Drug ApprovalOncology
  • Sino Biopharm achieved RMB17.57 billion in revenue for H1 2025, representing a 10.7% year-on-year increase, with innovative product sales reaching RMB7.8 billion, up 27.2%.
  • The company secured NMPA approval for two innovative products, Putanning and Anqixin, while maintaining a robust pipeline of 63 innovative drug candidates across four therapeutic areas.
  • Sino Biopharm completed the acquisition of LaNova Medicines in July 2025, gaining access to world-leading antibody discovery and ADC technology platforms to enhance R&D capabilities.
  • The company invested RMB3.19 billion in R&D during the period, representing 18.1% of revenue, supporting advancement of multiple Phase III trials including Lanifibranor for MASH treatment.

NewAmsterdam's Obicetrapib Receives EMA Validation for European Review Following Positive Phase 3 Results

Drug Approval
  • The European Medicines Agency has validated Marketing Authorization Applications for obicetrapib monotherapy and obicetrapib-ezetimibe combination therapy, marking a significant regulatory milestone for NewAmsterdam Pharma.
  • Phase 3 trials demonstrated clinically meaningful LDL-C reductions of 35-40% with obicetrapib monotherapy and approximately 50% with the combination therapy, both showing safety profiles comparable to placebo.
  • The partnership with Menarini Group provides NewAmsterdam with potential milestone payments up to €863 million and tiered double-digit royalties, while Menarini handles European commercialization responsibilities.
  • With cardiovascular disease causing 17.9 million deaths annually and many patients failing to reach LDL-C targets despite existing therapies, obicetrapib could address a significant unmet medical need.

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