Vamorolone

Santhera Announces NDA for Vamorolone in Duchenne Muscular Dystrophy Accepted and Granted Priority Review by China’s NMPA

Retrieved on: 
Wednesday, March 27, 2024
Diagnosis, NMPA, Patient, Ageing, NDA, Breakthrough, CDE, Duchenne muscular dystrophy, Therapy, Vamorolone, Safety, File, DMD, Neuromuscular disease, Pharmaceutical industry

Pratteln, Switzerland, March 27, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that the China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) which was submitted by Sperogenix Therapeutics, Santhera's specialized rare disease partner for China.

Key Points: 
  • Pratteln, Switzerland, March 27, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that the China National Medical Products Administration (NMPA) has accepted for priority review the new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) which was submitted by Sperogenix Therapeutics, Santhera's specialized rare disease partner for China.
  • The Center for Drug Evaluation (CDE) of the Chinese drug authority NMPA accepted the filing and granted priority review for vamorolone in DMD for patients aged 4 years and older which could, subject to a positive outcome, lead to approval by Q1 2025.
  • Duchenne muscular dystrophy is a rare neuromuscular disease affecting about 70,000 patients in China.
  • The submission is further supported by a study which investigated the pharmacokinetic parameters of vamorolone in healthy adult Chinese volunteers.

Santhera’s Partner Catalyst Pharmaceuticals Launches AGAMREE® (Vamorolone) in the United States

Retrieved on: 
Thursday, March 14, 2024
Catalyst Pharmaceuticals, Patient, Duchenne muscular dystrophy, Vamorolone, Food, FDA, Catalysis, DMD, Fine chemical

AGAMREE is the first DMD treatment approved across the U.S., EU and UK

Key Points: 
  • AGAMREE is the first DMD treatment approved across the U.S., EU and UK
    Pratteln, Switzerland, March 14, 2024 – Santhera Pharmaceuticals (SIX: SANN) is pleased to note the launch of AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in the United States (U.S.) by Catalyst Pharmaceuticals, Inc. (NASDAQ: CPRX), the Company’s commercialization partner for North America.
  • “Congratulations to our partner Catalyst on the launch of AGAMREE® in the United States.
  • According to the license agreement between the companies, first announced in June 2023 , Catalyst holds an exclusive North American license to commercialize AGAMREE for DMD and all potential future indications.
  • Under the agreement, Catalyst will pay Santhera sales-based milestones of up to USD 105 million as well as up to low-teen percentage royalties and will assume Santhera's corresponding third-party royalty obligations on AGAMREE sales in all indications in North America.

Catalyst Pharmaceuticals Announces AGAMREE® Now Commercially Available in the U.S. for the Treatment of Duchenne Muscular Dystrophy (DMD)

Retrieved on: 
Wednesday, March 13, 2024
Catalyst Pharmaceuticals, Steroid, Caregiver, Patient, Ageing, Duchenne muscular dystrophy, Hope, Fatality, Quality of life, Respiratory failure, Behavior, Vamorolone, FDA, DMD, Muscular dystrophy, Food, Pharmaceutical industry

CORAL GABLES, Fla., March 13, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company, today announced the U.S. commercial launch of AGAMREE® (vamorolone) oral suspension 40 mg/mL for the treatment of Duchenne Muscular Dystrophy ("DMD") in patients aged two years and older. Following the U.S. Food and Drug Administration ("FDA") approval on October 26, 2023, AGAMREE is now available by prescription and dispensed throughout the United States through a specialty pharmacy network.

Key Points: 
  • "Today, we proudly announced the U.S. commercial availability of AGAMREE, an innovative alternative steroid treatment for Duchenne Muscular Dystrophy.
  • Our Catalyst Pathways, a personalized program, is readily available to assist Duchenne Muscular Dystrophy patients and their families with the one‐on‐one education and financial support they need, thereby enhancing the access and affordability of AGAMREE for every patient.
  • For more information, caregivers and healthcare professionals can call 1-833-422-8259 or visit the Catalyst Pathways® website at www.yourcatalystpathways.com .
  • Duchenne Muscular Dystrophy, DMD, the most common form of muscular dystrophy, is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality.

Catalyst Pharmaceuticals to Present at MDA Clinical & Scientific Conference Details of Registry for Study of Long-Term Safety and Quality of Life in Duchenne Muscular Dystrophy Patients Treated with AGAMREE

Retrieved on: 
Tuesday, February 27, 2024
Vamorolone, Catalyst Pharmaceuticals, Physician, Quality of life, DMD, Duchenne muscular dystrophy, Conference, Doctor of Philosophy, Safety, Patient, Poster, Male, Episcopal conference, Pharmaceutical industry

The poster will detail the establishment of a multicenter, observational, prospective, longitudinal registry at approximately 25 U.S. sites.

Key Points: 
  • The poster will detail the establishment of a multicenter, observational, prospective, longitudinal registry at approximately 25 U.S. sites.
  • Investigators will assess the progress of 250 male patients who are at least 2 years old at week 12 and then every 12 months for up to five years.
  • The first interim analysis is expected in May 2025.
  • "We encourage physicians treating patients with DMD to consider enrolling eligible patients in this registry."

Catalyst Pharmaceuticals Announces Publication of Santhera Pharmaceutical’s VISION-DMD Vamorolone (AGAMREE®) Study Results in the Peer-Reviewed Journal Neurology

Retrieved on: 
Wednesday, February 21, 2024
Vamorolone, Catalyst Pharmaceuticals, NSAA, Safety, Prednisone, DMD, Growth, Duchenne muscular dystrophy, Neurology, Biomarker, BMI, Patient, LSM, JAMA Neurology, Pharmaceutical industry

Catalyst holds the exclusive rights to commercialize AGAMREE® (vamorolone) in North America.

Key Points: 
  • Catalyst holds the exclusive rights to commercialize AGAMREE® (vamorolone) in North America.
  • Based on the published study, Santhera has reported that the results of the VISION-DMD study support the long-term efficacy and safety profile of vamorolone and conclude that vamorolone was generally well tolerated, consistent with the 24-week study findings, as published previously in JAMA Neurology [2].
  • This study [VISION-DMD] was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone).
  • Improvements of motor outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment were maintained to 48 weeks of treatment.

Santhera Announces Publication of Efficacy, Safety and Tolerability Data with Vamorolone (AGAMREE®) in Patients with Duchenne Muscular Dystrophy in Neurology

Retrieved on: 
Wednesday, February 14, 2024
Vamorolone, NSAA, Safety, Prednisone, DMD, Growth, Duchenne muscular dystrophy, Neurology, European, Biomarker, BMI, Patient, Disease, LSM, JAMA Neurology, Pharmaceutical industry

“Vamorolone is a dissociative corticosteroid that selectively binds to the glucocorticoid receptor and has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD) [3].

Key Points: 
  • “Vamorolone is a dissociative corticosteroid that selectively binds to the glucocorticoid receptor and has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD) [3].
  • This study [VISION-DMD] was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone).
  • There was an increase in BMI after 24 weeks of treatment that then stabilized for both vamorolone groups.
  • Improvements of motor outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment were maintained to 48 weeks of treatment.

Santhera Launches AGAMREE® (Vamorolone) in Germany as First Market for the Treatment of Duchenne Muscular Dystrophy

Retrieved on: 
Monday, January 15, 2024
Medication package insert, Patient, European Medicines Agency, Prednisone, Steroid, EMA, Mutation, Disease, Vamorolone, Marketing, DMD, Duchenne muscular dystrophy, Growth, Pharmaceutical industry

“We are very happy that AGAMREE is now commercially available in Germany, the first country worldwide, for the treatment of DMD,” said Dario Eklund, CEO of Santhera.

Key Points: 
  • “We are very happy that AGAMREE is now commercially available in Germany, the first country worldwide, for the treatment of DMD,” said Dario Eklund, CEO of Santhera.
  • “This significant milestone represents Santhera’s commitment to fill the unmet need in DMD and provide a safe and effective treatment for DMD patients.
  • Now the time has come—with the new drug AGAMREE, patients suffering from Duchenne muscular dystrophy have access to a better tolerated alternative to steroids.
  • For more information about AGAMREE in the European Union, please see the Summary of Product Characteristics (SmPC) [1].

Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy in the United Kingdom

Retrieved on: 
Friday, January 12, 2024
Ageing, Patient, MHRA, Medication, MD, European Medicines Agency, Health care, Prednisone, Civil service commission, Dual-energy X-ray absorptiometry, European Commission, Steroid, Medicine, DXA, Spine, EMA, Mutation, Food, Safety, Vamorolone, FDA, Biomarker, DMD, Duchenne muscular dystrophy, Organic, Lead, Growth, Pharmaceutical industry

We are working towards making AGAMREE available to patients in the UK in the second half-year 2024, after NICE completes its pricing review.

Key Points: 
  • We are working towards making AGAMREE available to patients in the UK in the second half-year 2024, after NICE completes its pricing review.
  • Duchenne UK and our partner charities, Joining Jack and the Duchenne Research Fund, funded the early-stage clinical research to test vamorolone in patients, when no-one else would.
  • Santhera will continue to collect data to further characterize the long-term effectiveness and the broader safety differentiation of vamorolone.
  • This makes AGAMREE the first and only medicinal product fully approved in the EU and UK for DMD, and the first treatment approved for the treatment of DMD all three geographies.

Human medicines European public assessment report (EPAR): Agamree, Vamorolone, Date of authorisation: 14/12/2023, Status: Authorised

Retrieved on: 
Friday, January 5, 2024
Medical Subject Headings, Marketing, Cushing's syndrome, Cytokine, Agency, Health, INN, Risk, EMA, Muscular dystrophy, International, Duchenne muscular dystrophy, Adrenal crisis, Ageing, Language, Mouth, Inflammation, Vamorolone, European Medicines Agency, DMD, Face, Growth, Select, Patient, Bruise, Safety, Vomiting, IIA, Medical device

Human medicines European public assessment report (EPAR): Agamree, Vamorolone, Date of authorisation: 14/12/2023, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Agamree, Vamorolone, Date of authorisation: 14/12/2023, Status: Authorised

Santhera’s AGAMREE® (Vamorolone) Approved in the European Union for the Treatment of Duchenne Muscular Dystrophy

Retrieved on: 
Monday, December 18, 2023
Patient, Food, EMA, FDA, Catalyst Pharmaceuticals, European, Duchenne muscular dystrophy, Prednisone, Dual-energy X-ray absorptiometry, DXA, Mutation, Vamorolone, European Medicines Agency, Spine, DMD, Biomarker, Growth, Committee, Safety, Organization, Food and Drug Administration, CHMP, Pharmaceutical industry

In addition, patients who switched from a standard of care corticosteroid to AGAMREE maintained the efficacy benefit while recovering their growth and bone health.

Key Points: 
  • In addition, patients who switched from a standard of care corticosteroid to AGAMREE maintained the efficacy benefit while recovering their growth and bone health.
  • Santhera will continue to collect data to further characterize the long-term effectiveness and the broader safety differentiation of vamorolone.
  • Santhera announced on October 27, 2023 that the FDA approved AGAMREE oral suspension 40 mg/ml in the United States for the treatment of DMD in patients 2 years of age and older.
  • Catalyst Pharmaceuticals holds an exclusive license for AGAMREE in North America and plans to launch the product in the U.S. in Q1-2024.