Muscle weakness

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawn

Retrieved on: 
Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Mitochondrial encephalomyopathy, Headache, DSM-IV codes, Lactic acidosis, Weakness, MELAS, Cell, Disease, Seizure, Paratriathlon, Patient, Committee, Knowledge, Mitochondrion, Regulation, EMA, IRIS, European, COMP, Sponsor, European Commission, Safety, Muscle weakness, Pain, Vomiting, Marketing, Movement, Medicine, Pharmaceutical industry

Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawn

Key Points: 


Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawn

Phathom Pharmaceuticals Launches Direct-to-Consumer Campaign, “VOQUEZNA Can Kick Some Acid”

Retrieved on: 
Tuesday, March 26, 2024
Connected, HIV-1, Amazon Prime Video, Breast milk, Face, Hypoesthesia, Patient, Chronic pain, Clostridioides, Heartburn, Lymph, Magnesium, Diarrhea, Myalgia, Spine, Allergy, Muscle weakness, Vonoprazan, Stomach, Vitamin, Dizziness, Potassium, Hip, Risk, Tongue, Chills, DTC, Safety, Food, PPI, Rash, Food and Drug Administration, GERD, Enzyme inhibitor, Skin, Esophagitis, HIV, Rilpivirine, Kidney, FDA, Esophagus, B-12, Weakness, Infection, Indigestion, Smart TV, Seizure, Inflammation, Polyp, Calcium, Extrapyramidal system, Bone fracture, Nausea, Spasm, Urine, Fever, Clostridioides difficile, Bleeding, Blood, Wrist, Blister, Vitamin B12, Human, Medicine, Pharmaceutical industry

FLORHAM PARK, N.J., March 26, 2024 (GLOBE NEWSWIRE) -- Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal (GI) diseases, today announces the launch of its new broadcast ad and full-scale, Direct-to-Consumer (DTC) campaign, “VOQUEZNA Can Kick Some Acid,” to raise awareness of its powerful first-in-class treatment and encourage people to speak to their doctor about this new treatment option. VOQUEZNA (vonoprazan) is indicated for the healing and maintenance of healing of all severities of Erosive Esophagitis (EE), also referred to as Erosive GERD, and for the relief of related heartburn. VOQUEZNA represents the first major innovation to the U.S. Erosive GERD market in over 30 years.1

Key Points: 
  • The campaign will also be featured on consumer-facing platforms across Facebook, Instagram, waiting room TVs in doctor offices, and digital banner ads.
  • “Phathom is excited to launch our first campaign directly to, and for, the people whose lives we strive to improve every day.
  • To view a video of the “VOQUEZNA Can Kick Some Acid” commercial, click here.
  • Talk with your healthcare provider about the possibility of fundic gland polyps if you have been on VOQUEZNA for a long time.

STEALTH BIOTHERAPEUTICS ANNOUNCES FDA ACCEPTANCE OF NEW DRUG APPLICATION FOR ELAMIPRETIDE FOR THE TREATMENT OF BARTH SYNDROME

Retrieved on: 
Monday, April 8, 2024
Friends, New Drug Application, Elamipretide, Patient, Barth syndrome, NDA, Syndrome, Ultra, Quality of life, Safety, Muscle weakness, Stealth, Fatigue, FDA, Food, Medicine, Pharmaceutical industry

NEEDHAM, Mass., April 8, 2024 /PRNewswire/ -- Stealth BioTherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration ("FDA") has accepted for filing its New Drug Application ("NDA") for elamipretide for the treatment of Barth syndrome. The NDA is supported by the positive data from the SPIBA-001 Phase 3 Natural History Control Study and additional supporting efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020. 

Key Points: 
  • Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020.
  • The FDA indicated that it is currently planning to hold an advisory committee meeting to discuss the application.
  • The application was assigned a standard review designation, which the Company has asked FDA to reconsider.
  • Elamipretide is also in development for primary mitochondrial myopathy, with pivotal data from the fully-enrolled Phase 3 NuPOWER trial expected in late 2024.

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Civil service commission, Wasting, Diagnosis, Atrophy, Mutation, B cell, Weakness, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Death, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, Safety, European Commission, Muscle weakness, Marketing, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Key Points: 


Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Retrieved on: 
Tuesday, April 9, 2024
Community, Eurostat, Civil service commission, Diagnosis, U1 spliceosomal RNA, Capsid, Gene, B cell, Cell, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Protein, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, European Commission, Safety, Muscle weakness, Blood, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Key Points: 


Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Civil service commission, Eating, Diagnosis, Azathioprine, Disease, Dermatomyositis, Risk, Rash, Inflammation, Patient, Committee, Knowledge, Tissue, Regulation, EMA, IRIS, Mouth, European, Skin, COMP, Heart, Sponsor, European Commission, Immune system, Safety, Muscle weakness, Skeletal muscle, Stomach, Marketing, Medicine, Pharmaceutical industry

Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn

Key Points: 


Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn

NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US

Retrieved on: 
Friday, March 22, 2024
MuSK protein, Science Translational Medicine, Charcot–Marie–Tooth disease, GMG, Patient, Myasthenia gravis, IND, Spinal muscular atrophy, Acetylcholine receptor, Safety, Muscle weakness, Fatigue, FDA, Food, Pharmaceutical industry

The Phase 2b clinical trial is a dose range-finding, double-blind, placebo-controlled study of NMD670, a twice daily oral muscle-targeted therapy, in gMG patients experiencing persistent symptoms or fluctuating symptoms despite treatment on current standard of care.

Key Points: 
  • The Phase 2b clinical trial is a dose range-finding, double-blind, placebo-controlled study of NMD670, a twice daily oral muscle-targeted therapy, in gMG patients experiencing persistent symptoms or fluctuating symptoms despite treatment on current standard of care.
  • The trial will take place in both US and European clinical sites.
  • Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: “This is an important milestone for NMD Pharma, enabling us to progress our lead development candidate NMD670 into a Phase 2b trial in the US in generalized myasthenia gravis patients with AChR and MuSK positive antibodies.
  • It follows positive Phase 1/2a data which provided proof-of-mechanism and where clinically and statistically significant effects were seen in patients.

NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US

Retrieved on: 
Friday, March 22, 2024
MuSK protein, Science Translational Medicine, Charcot–Marie–Tooth disease, GMG, Patient, Myasthenia gravis, IND, Spinal muscular atrophy, Acetylcholine receptor, Safety, Muscle weakness, Fatigue, FDA, Food, Pharmaceutical industry

The Phase 2b clinical trial is a dose range-finding, double-blind, placebo-controlled study of NMD670, a twice daily oral muscle-targeted therapy, in gMG patients experiencing persistent symptoms or fluctuating symptoms despite treatment on current standard of care.

Key Points: 
  • The Phase 2b clinical trial is a dose range-finding, double-blind, placebo-controlled study of NMD670, a twice daily oral muscle-targeted therapy, in gMG patients experiencing persistent symptoms or fluctuating symptoms despite treatment on current standard of care.
  • The trial will take place in both US and European clinical sites.
  • Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: “This is an important milestone for NMD Pharma, enabling us to progress our lead development candidate NMD670 into a Phase 2b trial in the US in generalized myasthenia gravis patients with AChR and MuSK positive antibodies.
  • It follows positive Phase 1/2a data which provided proof-of-mechanism and where clinically and statistically significant effects were seen in patients.

NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine

Retrieved on: 
Thursday, March 21, 2024
MuSK protein, Exercise, Science Translational Medicine, Charcot–Marie–Tooth disease, NMD, Patient, Brain cell, Human, Myasthenia gravis, Spinal muscular atrophy, Acetylcholine receptor, Facial muscles, QMG, Myasthenia, Safety, Aarhus University, Muscle weakness, Fatigue, Paper, Neuromuscular disease, Pharmaceutical industry

NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.

Key Points: 
  • NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.
  • NMD670 is a muscle-targeted therapy which aims to enhance the activation of skeletal muscle fibers and to improve muscle strength and endurance.
  • As a result, patients have impaired skeletal muscle function and often experience severe muscle weakness and fatigue.
  • Access the full paper in Science Translational Medicine online here:
    In October 2022, NMD Pharma announced positive topline data which established the first clinical proof-of-mechanism of NMD Pharma’s novel CIC-1 chloride channel inhibitor approach in patients impacted by MG.

NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine

Retrieved on: 
Thursday, March 21, 2024
MuSK protein, Exercise, Science Translational Medicine, Charcot–Marie–Tooth disease, NMD, Patient, Brain cell, Human, Myasthenia gravis, Spinal muscular atrophy, Acetylcholine receptor, Facial muscles, QMG, Myasthenia, Safety, Aarhus University, Muscle weakness, Fatigue, Paper, Neuromuscular disease, Pharmaceutical industry

NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.

Key Points: 
  • NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.
  • NMD670 is a muscle-targeted therapy which aims to enhance the activation of skeletal muscle fibers and to improve muscle strength and endurance.
  • As a result, patients have impaired skeletal muscle function and often experience severe muscle weakness and fatigue.
  • Access the full paper in Science Translational Medicine online here:
    In October 2022, NMD Pharma announced positive topline data which established the first clinical proof-of-mechanism of NMD Pharma’s novel CIC-1 chloride channel inhibitor approach in patients impacted by MG.