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Q3 2025 Biopharma Catalyst Monitor Highlights 18 Key FDA Decisions and Clinical Trial Milestones

Drug ApprovalRare Disease
  • A new quarterly report identifies 18 significant biopharma catalyst events expected in Q3 2025, including FDA approval decisions for multiple companies across diverse therapeutic areas.
  • Key regulatory milestones include potential approvals for Ascendis Pharma's Skytrofa for adult growth hormone deficiency, PTC Therapeutics' sepiapterin for phenylketonuria, and Lenz Therapeutics' presbyopia treatment LNZ100.
  • The report, based on key opinion leader interviews, also covers ongoing clinical trials for Eli Lilly's orforglipron for obesity and aTyr Pharma's efzofitimod for pulmonary sarcoidosis.
  • Industry experts will assess the commercial potential of these treatments and their impact on existing therapeutic landscapes across multiple disease areas.

Iovance Biotherapeutics Shares Surge 14% on Amtagvi FDA Approval and New CEO Appointment

Drug ApprovalLeadership Change
  • Iovance Biotherapeutics shares surged 14.46% in pre-market trading following FDA approval of its cancer treatment Amtagvi and positive clinical trial results.
  • The company appointed Corleen Roche as new CEO, bringing extensive biotech financial management and commercial launch experience to enhance strategic direction.
  • Despite reducing guidance, Iovance projects $275 million in product revenue for 2025, primarily driven by Amtagvi sales.
  • Recent financial results showed $49.32 million in revenue with a 21.7% gross margin, though operational expenses exceeded $121 million.

Lantheus Completes $789 Million Acquisition of Life Molecular Imaging, Gains FDA-Approved Alzheimer's Diagnostic Agent

Drug Approval
  • Lantheus Holdings completed its $789 million acquisition of Life Molecular Imaging on July 22, 2025, gaining access to Neuraceq, an FDA-approved F-18 PET imaging agent for detecting beta-amyloid plaques in Alzheimer's disease patients.
  • The transaction provides Lantheus with robust Alzheimer's disease radiodiagnostic commercial infrastructure, advanced R&D capabilities, and an established international footprint across multiple markets.
  • Former Life Molecular Imaging CEO Dr. Ludger Dinkelborg joined Lantheus as Head of Research and Development on August 1, 2025, bringing proven leadership in advancing innovative radiopharmaceuticals.
  • Neuraceq received FDA approval on June 23, 2025, for positron emission tomography of the brain to estimate amyloid beta neuritic plaque density in adults with cognitive impairment.

FDA Clears Takeda's HyHub Devices to Streamline At-Home HYQVIA Immunoglobulin Therapy

Drug Approval
  • The FDA has granted 510(k) clearance to Takeda's HyHub and HyHub Duo devices, which enable needle-free transfer of HYQVIA's dual-vial components for patients aged 17 and older.
  • The devices reduce preparation steps by up to 50% compared to traditional pooling bag methods and minimize the need for ancillary supplies during infusion preparation.
  • HYQVIA is approved for treating primary immunodeficiency in adults and children as young as two, and as maintenance therapy for chronic inflammatory demyelinating polyneuropathy in adults.
  • The devices will be available at no additional cost to patients starting in the second half of fiscal year 2025, with a CE Mark application under review in the European Union.

Long-Term Tolerability and Safety of HYQVIA/HyQvia in CIDP

NCT02955355CompletedPhase 3
Baxalta now part of Shire
Posted 11/14/2016

A Study of HyQvia and Gammagard Liquid (Kiovig) in Adults With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

NCT02549170CompletedPhase 3
Baxalta now part of Shire
Posted 12/15/2015

NICE Approves First-in-Class Birch Pollen Immunotherapy for Severe Hayfever on NHS

Drug Approval
  • NICE has recommended Itulazax 12 SQ-Bet, an under-the-tongue immunotherapy tablet containing birch pollen extract, for NHS use in England for patients with severe hayfever unresponsive to conventional treatments.
  • The treatment works by training the immune system to tolerate tree pollen through three years of daily use, offering long-term relief rather than symptom management.
  • Approximately 27,000 people in England are expected to benefit from this recommendation, with birch pollen allergy affecting about 25% of hayfever sufferers.
  • The NHS will provide the treatment within three months of NICE publishing its final guidance, marking a significant advancement for the UK allergy community.

ALK's Tree Pollen Tablet ITULAZAX Receives NICE Recommendation for UK NHS Reimbursement

Drug Approval
  • ALK's tree pollen tablet ITULAZAX has been recommended by NICE for use in the UK health system, making it eligible for general reimbursement through the NHS in England, Wales, and Northern Ireland.
  • This marks ALK's second NICE approval for sublingual allergy immunotherapy products in 2025, following ACARIZAX approval in March, addressing significant underutilization of AIT in the UK compared to other European countries.
  • The approval comes as scientists warn of an early tree pollen season with higher counts, affecting approximately 16 million people with hay fever in the UK, including 4 million with moderate to severe tree pollen allergy.
  • NICE published Final Draft Guidance on July 21, 2025, with Final Guidance expected in August, after which NHS systems will be required to implement ITULAZAX in treatment practices.

CDSCO Releases New SEC Guidelines to Enhance Drug and Medical Device Approval Process

Drug Approval
  • The Central Drugs Standard Control Organisation (CDSCO) has released comprehensive Subject Expert Committees (SECs) Guidance Document Version 1.0 to streamline India's regulatory approval process for drugs and medical devices.
  • The new guidelines establish clear criteria for SEC members, requiring minimum 10 publications and 2:1 citation ratio, while mandating structured review processes with seven-day timelines for recommendations.
  • Applicants must now submit focused briefing materials limited to 20-25 slides five days before meetings, emphasizing data transparency and post-approval compliance requirements.
  • The guidelines clarify clinical trial waiver provisions for orphan drugs, pandemic treatments, and therapies approved in major regulatory jurisdictions including USA, UK, Japan, Canada, Australia, and EU.

Protalix BioTherapeutics Appoints Gilad Mamlok as New CFO to Drive Growth Strategy

Rare DiseaseDrug Approval
  • Protalix BioTherapeutics has appointed Gilad Mamlok as Senior Vice President and Chief Financial Officer, effective August 24, 2025, succeeding Eyal Rubin.
  • Mamlok brings three decades of experience in healthcare and technology companies, including roles at TytoCare, Sol-Gel Technologies, and Given Imaging.
  • The appointment comes as Protalix continues to advance its pipeline of plant cell-based therapeutic proteins, including approved products Elfabrio and taliglucerase alfa.
  • The company's proprietary ProCellEx platform represents the first FDA-approved protein production system using plant cell-based suspension expression technology.

Congressional Budget Office Warns Trump's Proposed NIH Cuts Could Reduce Drug Approvals by 4.5%

Drug Approval
  • The Congressional Budget Office warns that President Trump's proposed $18 billion NIH funding cut could reduce new drug approvals by 4.5%, equivalent to approximately two fewer drugs per year.
  • A modest 10% reduction in NIH preclinical research funding would decrease phase 1 drug candidates by one in the first decade, nine in the second, and twenty in the third decade.
  • Proposed FDA budget cuts and extended review times could further compound the impact, with nine-month delays potentially reducing drug approvals by an additional 2% annually.
  • The NIH has already rescinded over $3.2 billion in research funding in 2025, terminating 2,548 grants nationwide according to Grant Watch tracking data.

FDA Approves Osilodrostat for Second-Line Cushing Syndrome Treatment as Pipeline Advances

Drug ApprovalRare Disease
  • The FDA approved osilodrostat (Isturisa) as a second-line treatment for adults with endogenous Cushing syndrome in April 2025, expanding treatment options for patients unable to undergo surgery.
  • Relacorilant demonstrated significant cardiometabolic benefits in Phase III trials, showing notable blood pressure reductions of -10.1 mm Hg systolic and -6.3 mm Hg diastolic compared to placebo increases.
  • Multiple companies including Corcept Therapeutics, Crinetics Pharmaceuticals, and AstraZeneca are developing novel therapies with over 4 investigational treatments in various clinical phases.
  • The Cushing syndrome pipeline includes diverse therapeutic approaches targeting cortisol synthesis inhibition and glucocorticoid receptor modulation across oral and parenteral administration routes.

Study to Determine the Prevalence of Hypercortisolism in Patients With Type 2 Diabetes and Treatment With Korlym® (Mifepristone) (CATALYST)

NCT05772169CompletedPhase 4
Corcept Therapeutics
Posted 3/31/2023

A Study of the Efficacy and Safety of Relacorilant in Patients With Cortisol-Secreting Adrenal Adenomas

NCT04308590CompletedPhase 3
Corcept Therapeutics
Posted 7/27/2020

A Study of the Efficacy and Safety of Relacorilant in Patients With Endogenous Cushing Syndrome

NCT03697109CompletedPhase 3
Corcept Therapeutics
Posted 11/15/2018

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