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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  • Health Canada has released draft guidance that would eliminate the requirement for comparative Phase III clinical trials in biosimilar drug approvals, marking a significant policy reversal.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  • The consultation period for the draft guidance runs until September 8, 2025, with potential implementation representing a pivotal change in Canada's biosimilar regulatory landscape.

China Proposes 30-Day Review Pathway for Innovative Drug Clinical Trials Nationwide

Drug ApprovalRare Disease
  • China's NMPA has issued a draft announcement proposing to reduce clinical trial review times from 60 to 30 business days for qualifying innovative drugs nationwide.
  • The accelerated pathway applies to Registration Category I innovative drugs meeting specific criteria including nationally prioritized drugs, pediatric/rare disease programs, or global synchronized trials.
  • The proposal expands beyond the previous Beijing and Shanghai pilot program to cover all innovative drugs including chemical drugs, biologics, and traditional Chinese medicines across the country.
  • Public comments on the draft policy are due by July 16, 2025, as China continues to strengthen its position in global drug development with approximately one-third of multinational licensing deals now involving Chinese drugs.

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

NCT02290951CompletedPhase 1
Regeneron Pharmaceuticals
Posted 1/9/2015

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

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 Adult Participants With Follicular Lymphoma

NCT06091254Active, Not RecruitingPhase 3
Regeneron Pharmaceuticals
Posted 12/12/2023

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.

Supernus Pharmaceuticals Acquires Sage Therapeutics for Up to $795 Million, Securing Postpartum Depression Drug Zurzuvae

Drug Approval
  • Supernus Pharmaceuticals agreed to acquire Sage Therapeutics for up to $795 million, offering $8.50 per share in cash plus contingent value rights worth $3.50 per share based on sales milestones.
  • The deal significantly exceeds Biogen's rejected $469 million unsolicited offer from January 2025, providing Sage shareholders with a 36% premium over previous trading levels.
  • Supernus gains exclusive U.S. rights to Zurzuvae, the only FDA-approved oral treatment for postpartum depression, which generated $36.1 million in 2024 sales and $13.8 million in Q1 2025.
  • The acquisition allows Supernus to diversify its CNS portfolio beyond its existing Parkinson's disease and ADHD treatments, while positioning the company for significant growth if Zurzuvae reaches projected sales milestones.

Biologic Therapies Transform Respiratory Care as Clinicians Embrace Earlier, Precision-Based Treatment Strategies

Precision MedicineDrug Approval
  • Biologic therapies including dupilumab, benralizumab, and tezepelumab have revolutionized asthma management by enabling biomarker-driven treatment strategies beyond traditional allergen or eosinophil-based models.
  • Dupilumab's 2024 FDA approval for eosinophilic COPD represents the first biologic therapy for this condition, marking a significant milestone in a field where treatment had previously stagnated.
  • Clinicians are increasingly adopting earlier biologic intervention strategies to minimize steroid exposure and prevent long-term adverse outcomes, with pulmonologists now managing biologics more independently.
  • The recognition of asthma and COPD as heterogeneous diseases with overlapping inflammatory pathways is driving a shift toward personalized medicine approaches guided by biomarkers like eosinophils and FeNO.

FDA Delays KalVista's Sebetralstat Approval Decision Due to Resource Constraints

Drug Approval
  • The FDA has extended its review of KalVista's sebetralstat for hereditary angioedema by four weeks past the June 17 deadline due to heavy workload and limited resources.
  • Sebetralstat would become the first on-demand oral treatment for hereditary angioedema if approved, offering a convenient alternative to injectable therapies.
  • The delay represents the first instance directly attributed to FDA resource constraints following agency restructuring under the Trump administration.
  • KalVista maintains confidence in near-term approval as the FDA has not requested additional data or raised safety concerns.

FDA Delays Sebetralstat Approval Decision for Hereditary Angioedema Treatment Due to Resource Constraints

Drug ApprovalRare Disease
  • The FDA will not meet the June 17, 2025 PDUFA goal date for KalVista's sebetralstat NDA due to heavy workload and limited resources, with a decision expected within four weeks.
  • Sebetralstat represents a potential breakthrough as the first oral on-demand treatment for hereditary angioedema attacks in patients aged 12 and older.
  • The FDA has not requested additional data or raised safety concerns, with only labeling finalization remaining under review according to KalVista.
  • The delay affects patients with hereditary angioedema who currently rely on intravenous or subcutaneous treatments for managing painful and potentially life-threatening attacks.

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