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BrightSpring's Onco360 Pharmacy Selected as National Partner for Three New Cancer and Rare Disease Therapies

OncologyRare DiseaseDrug ApprovalTargeted Therapy
  • BrightSpring Health Services announced that its specialty pharmacy Onco360 has been selected as the national pharmacy partner for three newly approved therapies targeting advanced cancers and rare genetic disorders.
  • The partnership includes GOMEKLI for neurofibromatosis type 1 patients with plexiform neurofibromas, AVMAPKI FAKZYNJA CO-PACK for KRAS-mutated recurrent low-grade serous ovarian cancer, and IBTROZI for ROS1-positive non-small cell lung cancer.
  • Onco360 will provide comprehensive patient support including access, education, data management, and expert clinical guidance for these innovative treatments across all 50 states.

New Zealand Approves Over-the-Counter Melatonin and Prescription Psilocybin for Treatment-Resistant Depression

Drug Approval
  • New Zealand's MedSafe has approved melatonin for over-the-counter sale in pharmacies, making it available in doses up to 5mg for treating insomnia and jet lag.
  • Psilocybin will become available as an unapproved medicine for patients with treatment-resistant depression under the supervision of one experienced psychiatrist.
  • The psilocybin approval marks the first time the psychedelic compound will be legally available in New Zealand outside of clinical trials.
  • Both regulatory changes represent significant shifts in New Zealand's approach to alternative therapeutic options for sleep disorders and mental health conditions.

Bavarian Nordic Sells Priority Review Voucher for $160 Million Following Chikungunya Vaccine Approval

Drug ApprovalFDA Approval
  • Bavarian Nordic announced the sale of its Priority Review Voucher for $160 million, awarded following FDA approval of its chikungunya vaccine VIMKUNYA in February 2025.
  • The transaction is expected to close in Q3 2025 and will positively impact the company's EBITDA, with the National Institutes of Health receiving 20% of proceeds under a licensing agreement.
  • The sale reflects an uptick in PRV values in 2025, with recent transactions ranging from $150-160 million compared to the historical average of around $100 million.
  • Priority Review Vouchers reduce FDA drug review times from ten months to six months and are awarded for treatments addressing neglected tropical diseases, rare pediatric conditions, or material threat medical countermeasures.

Health Canada Approves Expanded Dosing and Pediatric Indication for FIRDAPSE in Lambert-Eaton Myasthenic Syndrome

Drug ApprovalRare Disease
  • Health Canada has approved Kye Pharmaceuticals' supplemental new drug submission for FIRDAPSE (amifampridine), allowing some patients with Lambert-Eaton myasthenic syndrome to benefit from an increased maximum daily dose of up to 100mg.
  • The expanded approval includes pediatric dosing guidance for patients 6 years of age and older, providing pediatric neurologists with a proven treatment option for children diagnosed with LEMS.
  • FIRDAPSE is a potassium channel blocker that works by increasing acetylcholine release at the neuromuscular junction, helping improve muscle function in patients with this rare autoimmune disorder.
  • The label update offers healthcare providers and patients greater flexibility in treatment regimens for managing LEMS, a condition characterized by muscle weakness and fatigue.

Canada Outpaces OECD Countries in New Medicine Launches Despite Rising Treatment Costs

Drug Approval
  • Canada launched more new medicines than the median for OECD countries in 2022-2023, according to the PMPRB's 9th annual Meds Entry Watch report.
  • Most new medicines entering the Canadian market carry high treatment costs exceeding $10,000 per year or $5,000 per 28-day cycle for oncology treatments.
  • Specialty medicines including biologics, orphan drugs, and cancer treatments represent a growing share of the new drug landscape in Canada.
  • The report analyzed 48 medicines receiving first-time market approval in 2022 and provided preliminary analysis of 63 medicines approved in 2023.

Health Canada Proposes Elimination of Phase III Clinical Trials for Biosimilar Approvals

Drug Approval
  • Health Canada has released draft guidance that would eliminate the requirement for comparative Phase III clinical trials in biosimilar drug approvals, marking a significant policy reversal.
  • The proposed changes would limit clinical requirements to comparative pharmacokinetic studies and optional pharmacodynamic evaluations, focusing instead on extensive analytical similarity data.
  • This regulatory shift aligns with similar moves by the European Medicines Agency and FDA discussions that have led some companies to terminate or minimize Phase III biosimilar trials.
  • The consultation period for the draft guidance runs until September 8, 2025, with potential implementation representing a pivotal change in Canada's biosimilar regulatory landscape.

Formycon Launches €50 Million Corporate Bond to Accelerate Biosimilar Portfolio Development

Drug Approval
  • Formycon AG announced plans to issue a four-year corporate bond with a target volume of €50 million to fund the development and expansion of its biosimilar product portfolio.
  • The unsecured floating rate bond carries an interest rate based on three-month EURIBOR plus a 7.0% to 7.5% margin, with proceeds supporting the company's transition from development to commercial focus.
  • The public offering will be available in Germany, Luxembourg, and Austria, with subscription periods running from June 18-30, 2025, marking Formycon's first use of debt capital financing.
  • The company currently has three FDA and EMA-approved biosimilars including FYB201/ranibizumab already marketed in Europe and the USA, with four additional candidates in development targeting a biosimilar market projected to reach $74 billion by 2030.

FDA Approves CSL's ANDEMBRY for Hereditary Angioedema Prevention with Novel Factor XIIa Targeting

Drug ApprovalMonoclonal AntibodyRare Disease
  • The FDA approved ANDEMBRY (garadacimab-gxii), the first and only prophylactic HAE treatment targeting factor XIIa, offering once-monthly dosing for patients 12 years and older.
  • Phase 3 VANGUARD trial data showed ANDEMBRY reduced HAE attacks by a median of more than 99% and a least squares mean of 89.2% compared to placebo, with 62% of patients remaining attack-free.
  • ANDEMBRY represents a breakthrough approach by inhibiting the top of the HAE cascade through factor XIIa targeting, providing sustained protection with convenient subcutaneous self-injection in 15 seconds or less.
  • CSL will launch ANDEMBRY commercially immediately with availability before the end of June, expanding treatment options for the approximately 1 in 50,000 people affected by this rare genetic disorder.

CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks

NCT04656418CompletedPhase 3
CSL Behring
Posted 1/27/2021

FDA Issues Second Complete Response Letter for Odronextamab in Relapsed/Refractory Follicular Lymphoma

Drug Approval
  • The FDA has issued a second complete response letter (CRL) for odronextamab's biologics license application, this time related to observations from an FDA site inspection at Catalent Indiana, LLC manufacturing facility.
  • Updated ELM-2 trial data with 28.3 months median follow-up demonstrate sustained efficacy with 80.5% overall response rate and 74.2% complete response rate in heavily pretreated follicular lymphoma patients.
  • The bispecific antibody received European Union approval in August 2024 for relapsed/refractory follicular lymphoma and diffuse large B-cell lymphoma after at least 2 prior therapies.
  • Safety profile remains consistent with previous reports, dominated by manageable cytokine release syndrome and infection-related adverse events requiring careful patient monitoring and prophylaxis.

Study to Investigate the Safety and Tolerability of Odronextamab in Patients With CD20+ B-Cell Malignancies

NCT02290951Active, Not RecruitingPhase 1
Regeneron Pharmaceuticals
Posted 1/9/2015

A Trial to Learn if Odronextamab is Safe and Well-Tolerated and How Well it Works Compared to Rituximab Combined With Different Types of Chemotherapy for Participants With Follicular Lymphoma

NCT06091254RecruitingPhase 3
Regeneron Pharmaceuticals
Posted 12/12/2023

A Study to Assess the Anti-Tumor Activity and Safety of Odronextamab in Adult Patients With B-cell Non-Hodgkin Lymphoma Who Have Been Previously Treated With Other Cancer Therapies

NCT03888105RecruitingPhase 2
Regeneron Pharmaceuticals
Posted 11/13/2019

Supernus Pharmaceuticals Acquires Sage Therapeutics for $795 Million, Strengthening Neuropsychiatry Portfolio

Drug Approval
  • Supernus Pharmaceuticals announced a definitive agreement to acquire Sage Therapeutics for $8.50 per share upfront plus contingent payments up to $3.50 per share, totaling up to $795 million.
  • The acquisition adds ZURZUVAE (zuranolone), the first and only FDA-approved oral treatment for postpartum depression, to Supernus' neuropsychiatry portfolio.
  • The deal is expected to be significantly accretive in 2026 with potential cost synergies of up to $200 million annually and strengthen Supernus' CNS discovery platforms.
  • ZURZUVAE generated $36.1 million in collaboration revenue for 2024, with Supernus receiving 50% of net revenue recorded by partner Biogen in the U.S.

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