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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.

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

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

FDA Approves EXZOLT, First Oral Treatment for Northern Fowl Mites in Poultry

Drug Approval
  • The FDA has approved EXZOLT (fluralaner oral solution), the first systemic oral treatment for northern fowl mites in chickens, offering a novel alternative to traditional topical sprays.
  • Penn State researchers conducted pivotal efficacy studies showing over 90% reduction in mite populations, demonstrating the treatment's effectiveness in both broiler breeders and layer chickens.
  • The innovative water-administered treatment targets the parasite's nervous system while maintaining a zero-day egg withdrawal period and 11-day meat withdrawal period.
  • EXZOLT addresses significant economic losses in poultry operations caused by northern fowl mites, which reduce weight gain, egg quality, and production in affected birds.

FDA Advisory Panel Rejects GSK's Blenrep Combination Therapies for Multiple Myeloma

Drug Approval
  • FDA advisers voted 5-3 against approving GSK's Blenrep in combination with bortezomib and dexamethasone for relapsed or refractory multiple myeloma patients.
  • The panel voted 7-1 against Blenrep combined with pomalidomide and dexamethasone, citing unfavorable benefit-risk profile for the proposed patient population.
  • FDA briefing documents expressed concerns about Blenrep's unclear benefit-risk profile, safety, tolerability, and appropriate dosages.
  • Multiple myeloma is the third most common blood cancer and is generally treatable but not curable.

FDA Authorizes Juul E-Cigarettes for Continued Market Sale After Multiyear Review

Drug Approval
  • The FDA has authorized Juul Labs to continue selling its tobacco and menthol-flavored e-cigarettes after determining they are "appropriate for the protection of public health."
  • The decision follows a multiyear regulatory review that began in 2022 when the FDA initially ordered Juul to remove its products from the market.
  • Juul provided evidence showing high rates of adult smokers switch from traditional cigarettes to its products, reducing exposure to deadly carcinogens.
  • The authorization makes Juul one of only two U.S. companies permitted to sell menthol-flavored vaping products to adult consumers.

FDA-Approved Rectal Cancer Drug Metric Fails to Predict Long-Term Survival, Tulane Study Finds

Drug ApprovalOncology
  • A Tulane University meta-analysis of 25 clinical trials involving nearly 12,000 rectal cancer patients found no statistical relationship between pathologic complete response (pCR) and overall survival.
  • The study challenges the FDA's 2012 decision to allow pharmaceutical companies to use tumor-free status post-therapy as a surrogate for overall survival in drug approvals.
  • Researchers warn that cancer drugs may be fast-tracked for approval without demonstrating meaningful long-term improvements over existing treatments.
  • The findings suggest that relying solely on pCR as an endpoint may lead to increased costs for drug companies investing in therapies that cannot guarantee improved survival rates.

Rectal Cancer And Pre-operative Induction Therapy Followed by Dedicated Operation. The RAPIDO Trial

NCT01558921Active, Not RecruitingPhase 3
University Medical Center Groningen
Posted 6/21/2011

Citizens' Petition Seeks FDA Accelerated Approval for NurOwn Stem Cell Therapy in ALS Based on Survival Data

Drug ApprovalReal World Evidence
  • A 309-page Citizens' Petition has been filed with the FDA seeking accelerated approval of NurOwn, a mesenchymal stem cell therapy for ALS, based on unprecedented survival and respiratory data from trial participants.
  • The petition presents compelling real-world evidence including 100% five-year survival in the Expanded Access Program versus 20% natural history (p-value ~0.000335), and median 7-year tracheostomy-free survival compared to 30 months in natural history.
  • Neurologist Dr. Danielle Geraldi-Samara documented clinical improvements in multiple NurOwn recipients, including patients who regained motor function and maintained respiratory independence years beyond typical ALS progression timelines.
  • The petition argues that the FDA's focus on primary endpoints overlooked the totality of evidence, including patient experiences and expert clinical observations that demonstrate NurOwn's efficacy in slowing disease progression.

Harrow Secures Exclusive US Rights to Samsung Bioepis Ophthalmology Biosimilars Portfolio

Drug Approval
  • Harrow has entered into a partnership with Samsung Bioepis to gain exclusive US commercial rights to two FDA-approved ophthalmology biosimilars, BYOOVIZ and OPUVIZ, following Biogen's termination of its commercialization agreement.
  • The biosimilars target the $9 billion retinal disease treatment market, offering 30-40% wholesale acquisition cost reduction compared to reference drugs LUCENTIS and EYLEA.
  • BYOOVIZ became the first ophthalmology biosimilar approved in the US in September 2021, while OPUVIZ received FDA approval in May 2024 for broader indications including diabetic retinopathy.
  • The transition is expected to drive Harrow's revenue past $280 million in 2025, with analysts projecting the company could capture 15-20% of the US retinal disease market within three years.

Vor Bio Appoints Telitacicept Development Expert Dr. Qing Zuraw as Chief Development Officer

Drug Approval
  • Vor Bio has appointed Dr. Qing Zuraw as Chief Development Officer, bringing over 25 years of experience in autoimmune disease clinical development.
  • Dr. Zuraw previously led telitacicept development at RemeGen across four indications, achieving regulatory approvals in China for SLE, generalized myasthenia gravis, and rheumatoid arthritis.
  • The appointment positions Vor Bio to advance telitacicept through Phase 3 clinical development and global commercialization for autoantibody-driven conditions.
  • Dr. Zuraw secured multiple FDA designations for telitacicept including Fast Track, Breakthrough Therapy, and Orphan Drug status across various indications.

AAV Gene Therapy Pipeline Expands with 180+ Companies Developing 250+ Treatments Despite Recent Setbacks

Drug Approval
  • Over 180 companies are actively developing more than 250 AAV-based gene therapies, representing a robust pipeline for treating genetic disorders including hemophilia, spinal muscular atrophy, and inherited retinal diseases.
  • Recent FDA approvals include Roctavian for severe hemophilia A and Elevidys for Duchenne muscular dystrophy, demonstrating the therapeutic potential of AAV vectors in addressing rare genetic conditions.
  • The field has experienced notable setbacks, including Pfizer's termination of its hemophilia A partnership and a patient death linked to Elevidys treatment, highlighting ongoing safety challenges.
  • Leading pipeline candidates include DTX401 for glycogen storage disease, SRP-9003 for limb-girdle muscular dystrophy, and several innovative therapies targeting retinal diseases and cardiovascular conditions.

22nd Century Group Expands VLN Reduced-Nicotine Cigarette Partnerships Ahead of FDA Mandate

Drug Approval
  • 22nd Century Group is partnering with multiple tobacco brands including Smoker Friendly and Pinnacle to expand availability of its VLN reduced-nicotine cigarettes containing 95% less nicotine than conventional products.
  • The FDA's proposed nicotine reduction mandate would cap combustible tobacco products at 0.7 mg nicotine per gram of tobacco, with VLN products already meeting this standard at 0.5 mg per gram.
  • Clinical trials demonstrate VLN cigarettes reduce nicotine cravings by 33% and increase cessation success by 50% compared to conventional cigarettes, with no compensatory smoking behavior observed.
  • The FDA estimates the proposed standard could prevent 48 million youth and young adults from initiating smoking by 2100, representing a significant public health intervention.

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