Fenfluramine

Stoke Therapeutics Announces Landmark New Data That Support the Potential for STK-001 to be the First Disease-Modifying Medicine for the Treatment of Patients with Dravet Syndrome

Retrieved on: 
Monday, March 25, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Genetics, Clinical Trials, Diagnosis, Neurology, Socialization, Communication, Disease, Seizure, Patient, Fenfluramine, University, OLE, Behavior, Cognition, Therapy, Natural history, FAES, Dravet syndrome, Movement, Pediatrics, Food, Medicine, Pharmaceutical industry

Data from these studies showed clinically meaningful effects, including substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior that support the potential for disease modification.

Key Points: 
  • Data from these studies showed clinically meaningful effects, including substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior that support the potential for disease modification.
  • These improvements were observed among a highly refractory group of patients who were already taking the best available anti-seizure medicines.
  • Today, the Company also announced clearance from the U.S. Food and Drug Administration (FDA) that allows patients to receive three doses of 70mg followed by continued dosing at 45mg.
  • “STK-001 is the first medicine in development to demonstrate substantial and durable reductions in seizure frequency and improvements in multiple measures of cognition and behavior.

Enveric Biosciences Unveils Library of Preclinical Compounds Across Multiple Distinct Molecule Classes Targeting Mental Health Disorders

Retrieved on: 
Wednesday, February 21, 2024
Biotechnology, Mental Health, Health, Pharmaceutical, Clinical Trials, Therapy, Addiction, Paroxetine, Anxiety, DMT, Mental health, SP, Imipramine, Artificial intelligence, Tri, Serotonin, BPP, Sleep, MT1, HTR, Aripiprazole, Fenfluramine, MDMA, Trazodone, Human, Mammal, Patient, Hallucination, Melatonin, DSM-IV codes, Plasma, ADHD, Literature, Research, Nefazodone, Mouse, Depression, Pharmaceutical industry

“As a result, Enveric now possesses what we believe to be one of the most diverse portfolios of potential drug molecules for the treatment of key, underserved mental health and neurological disorders.

Key Points: 
  • “As a result, Enveric now possesses what we believe to be one of the most diverse portfolios of potential drug molecules for the treatment of key, underserved mental health and neurological disorders.
  • Enveric SNDRI compounds also demonstrate distinct additional serotonin receptor binding profiles that bear similarities to those of the antidepressant Nefazodone and the anxiolytic Buspirone.
  • Enveric NSRI compounds show strong binding to SERT, as well as to the 5-HT1A receptor, known to be a therapeutically valuable target.
  • For more information about Enveric’s novel preclinical compounds spanning multiple, distinct classes of molecules, please visit: https://www.enveric.com/psybrary .

Orphan designation: Fenfluramine hydrochloride Treatment of CDKL5 deficiency disorder, 20/03/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, CDKL5, CDKL5 deficiency disorder, Fenfluramine, Committee, EMA, Pharmaceutical industry

EU/3/23/2768 - orphan designation for treatment of CDKL5 deficiency disorder

Key Points: 
  • EU/3/23/2768 - orphan designation for treatment of CDKL5 deficiency disorder
    Fenfluramine hydrochloride
    OrphanHuman
    UCB Pharma
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Acino significantly expands presence and capabilities in Latin America through acquisition of M8 Pharmaceuticals

Retrieved on: 
Wednesday, September 20, 2023
Interim, Gastroenterology, Stifel, Marketing, History, Credit Suisse, CNS, Cooley LLP, Acquisition, Patient, PWC, Hematology, Growth, Rare, Caregiver, Fine chemical, Pharmaceutical industry, M8, Fenfluramine

Through this acquisition, Acino will enter the two largest pharmaceutical markets in Latin America, significantly expanding its geographic footprint and strengthening its position in the region, providing a more powerful and comprehensive offering for healthcare professionals and patients.

Key Points: 
  • Through this acquisition, Acino will enter the two largest pharmaceutical markets in Latin America, significantly expanding its geographic footprint and strengthening its position in the region, providing a more powerful and comprehensive offering for healthcare professionals and patients.
  • M8, a Montreux Growth Partners portfolio company, has a highly successful record of signing exclusive licensing agreements with large pharmaceutical and biotech companies for the rights to market and commercialize products in Latin America.
  • M8 brings a portfolio of well-known proven brands and innovative products, a best-in-class deal-making platform and an outstanding reputation among its partners.
  • Andrew Bird, Interim CEO at Acino commented: “This is a transformative deal for Acino, significantly expanding our presence in Latin America and delivering against our overall strategy and stated intention to increase patient’s access to high-quality pharmaceuticals and diversify our footprint in key high-growth markets.

Ozempic is in the spotlight but it's just the latest in a long and strange history of weight-loss drugs

Retrieved on: 
Tuesday, August 8, 2023
Exercise, Injection, Seizure, Amgen, Letter, Endocrinology, Human, Food, Obesity, Industry, Diet, US Food Sovereignty Alliance, Diabetes, Ephedra, Leptin, Stroke, Phentermine, Splenic infarction, Hope, Psychotherapy, Research, Appetite, History, Diarrhea, Due, Sale, Movement, Hormone, Science, Athlete, US, Dietary supplement, Patient, Time, Poisoning, Amphetamine, Novo Nordisk, Overweight, Hypothalamus, Pleasure, Fenfluramine, Nausea, Mouse, Growth, Knowledge, Health, Death, Safety, Social environment, Social media, Cosmetics, Canning, Birth control, Pharmaceutical industry, Drug, Nightclub, Semaglutide

That’s been the holy grail of weight-loss ever since 19th century English undertaker and weight-loss celebrity William Banting’s 1863 Letter on Corpulence spruiked his “miraculous” method of slimming down.

Key Points: 
  • That’s been the holy grail of weight-loss ever since 19th century English undertaker and weight-loss celebrity William Banting’s 1863 Letter on Corpulence spruiked his “miraculous” method of slimming down.
  • Since then, humans have tried many things – diet, exercise, psychotherapy, surgery – to lose weight.
  • But time and again we return to the promise of a weight-loss drug, whether it’s a pill, injection, or tonic.

Ozempic is a recent arrival

    • Ozempic and its sister drug Wegovy, both manufactured by Novo Nordisk, are the latest offerings in a long history of drug treatments for people who are overweight.
    • This has helped drive a shortage of Ozempic for diabetes treatment.

From ‘gland treatment’ to amphetamines

    • For example, organotherapy (gland treatment) was hugely popular in the 1920s to 1940s.
    • Doctors prescribed overweight people extracts of animal glands – either eaten raw or dried in pill form or injected – to treat their supposedly “sluggish glands”.
    • Amphetamines were first used as a nasal decongestant in the 1930s, but quickly found a market for weight-loss.
    • Amphetamines too, fell from treatment use in the 1970s with Nixon’s “war on drugs” and recognition they were addictive.

Another decade, another drug

    • For example, the popular diet drug of the 1980s and 90s was fen-phen, which contained appetite suppressants fenfluramine and phentermine.
    • And as history recognises, multiple complexities can combine to push a drug into popularity or damn it to history’s rubbish bin.
    • One noticeable contrast with past diet drug experiences is that now, many people are happy to talk about using Ozempic.
    • It seems to be increasingly socially acceptable to use a drug to achieve weight-loss for primarily aesthetic reasons.

Our enduring search for weight-loss drugs

    • Ozempic is predicted to earn Novo Nordisk US$12.5 billion this year alone, but it’s not just industry interests stoking this enduring desire for weight-loss drugs.
    • Patients on an endless cycle of dieting and exercise want something more convenient, with a more certain outcome.
    • It is no wonder demand for weight-loss drugs continues to soar.

UCB Showcases Strength and Depth of Neurology Portfolio at 75th American Academy of Neurology (AAN) Annual Meeting

Retrieved on: 
Thursday, April 20, 2023
Epilepsy, Abstract, LGS, AAN, SC, GMG, Lennox–Gastaut syndrome, DSM-IV codes, American Academy, Seizure, Summer Street Bridge disaster, UCB, RAISE, Fenfluramine, Boston Convention and Exhibition Center, Neonatal Fc receptor, GTA MyCar, Safety, Pharmaceutical industry, MD, Neurology

BRUSSELS and ATLANTA, April 20, 2023 /PRNewswire/ -- UCB will showcase data from its expansive and innovative neurology portfolio at the 75th American Academy of Neurology (AAN) Annual Meeting, April 22-27, 2023.

Key Points: 
  • BRUSSELS and ATLANTA, April 20, 2023 /PRNewswire/ -- UCB will showcase data from its expansive and innovative neurology portfolio at the 75th American Academy of Neurology (AAN) Annual Meeting, April 22-27, 2023.
  • The company will present 12 abstracts, including two oral presentations, across generalized myasthenia gravis (gMG), Lennox-Gastaut syndrome (LGS), and focal (partial) epileptic seizures.
  • In gMG, presentations will expand on the results from the MycarinG and RAISE studies1,2.
  • Charl van Zyl, Executive Vice President Neurology and Head of EU/International Markets, UCB, said: "Our presentations at AAN reinforce the strength and depth of our expanding rare disease pipeline and portfolio.

Stoke Therapeutics Highlights Strategic Priorities and Anticipated Milestones for 2023

Retrieved on: 
Monday, January 9, 2023
Science, Biotechnology, Research, Pharmaceutical, Optical, Health, Genetics, Clinical Trials, Disease, Seizure, Fenfluramine, Gene expression, ADOA, University of California, San Francisco Fetal Treatment Center, University, Therapy, Caregiver, Stoke, Quality of life, FDA, Dravet syndrome, Health, Pediatrics, Completeness, Conference, Central nervous system, Webcast, Patient, Neurology, Syndrome, CTA, Eye, Animal, Pharmaceutical industry

Data from patients treated with multiple doses of 70mg are expected in the second half of 2023.

Key Points: 
  • Data from patients treated with multiple doses of 70mg are expected in the second half of 2023.
  • Clinical Trial Application (CTA) submission in the UK for planned Phase 1/2 study in patients with ADOA is expected in the first half of 2023.
  • Stoke ended the third quarter of 2022 with $252.2 million in cash, cash equivalents, marketable securities, and restricted cash anticipated to fund operations into 2025.
  • Stoke’s Presentation at the 41st Annual J.P. Morgan Healthcare Conference
    Dr. Edward M. Kaye will discuss Stoke’s strategic priorities and key milestones in a presentation at the 41st Annual J.P. Morgan Healthcare Conference on Tuesday, January 10, 2023, at 2:15 p.m.

Stoke Therapeutics Presents Data from a Combined Interim Analysis of the Phase 1/2a MONARCH and ADMIRAL Studies of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2022 Annual Meeting

Retrieved on: 
Friday, December 2, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Linda, Safety, International League Against Epilepsy, Intellectual disability, Pharmacokinetics, Fenfluramine, DS, Patient, Adolescence, Language, Hospital, AES, Phase, Caregiver, Quality of life, Associate, Movement, Therapy, EMA, Cognition, SUDEP, Development of the nervous system, Lurie Children's Hospital, Dravet syndrome, Seizure, Even and odd functions, Syndrome, Autonomic nervous system, Helen Cross, Disease, Stoke, Annual general meeting, Diagnosis, Death, Ann, MB, Sleep, Medicine, SCN1A, Government budget balance, ASO, DMPK, OLE, Plasma, Prognosis, American Epilepsy Society, Observational study, FDA, Senior, Feinberg School of Medicine, Neurology, European Journal of Paediatric Neurology, Risk, Cerebrospinal fluid, Pharmaceutical industry, Vaccine, Medical imaging, Pediatric nursing, BioAnalyt, Epilepsy

A total of seven posters will be presented at the American Epilepsy Society (AES) 2022 Annual Meeting, December 2-6.

Key Points: 
  • A total of seven posters will be presented at the American Epilepsy Society (AES) 2022 Annual Meeting, December 2-6.
  • Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures beginning within the first year of life.
  • STK-001 represents an entirely new approach to treatment, one that aims to treat the syndrome, not just the seizures.
  • Topline data from a combined interim analysis of the Phase 1/2a MONARCH and ADMIRAL studies showed single and multiple doses of STK-001 up to 45mg were well-tolerated.

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates

Retrieved on: 
Monday, November 14, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Even and odd functions, Brain, Epilepsy, Sleep, Autonomic nervous system, Safety, Fenfluramine, Disease, Risk, License, Cognition, Plasma, SCN1A, Targeted molecular therapy for neuroblastoma, Dravet syndrome, OLE, Death, Research, Government budget balance, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Seizure, CSF, Diagnosis, TANGO, EMA, Patient, FDA, Language, United Kingdom, Intellectual disability, Prognosis, Therapy, Caregiver, Quality of life, Pharmacokinetics, Collaboration, ID, Asos, Protein, RNA, Food, American Epilepsy Society, Cerebrospinal fluid, ASO, Medicine, Haploinsufficiency, SUDEP, Movement, Pharmaceutical industry, Medical imaging, Coffee, Reinsurance, Animal, Vaccine, Stoke

MONARCH and ADMIRAL are multi-center, Phase 1/2a studies of children and adolescents who have an established diagnosis of Dravet syndrome.

Key Points: 
  • MONARCH and ADMIRAL are multi-center, Phase 1/2a studies of children and adolescents who have an established diagnosis of Dravet syndrome.
  • Today, the Company provided topline results from the ongoing Phase 1/2a MONARCH and ADMIRAL studies of STK-001.
  • Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.
  • Using Stokes proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels.

Epilepsia Publishes Interim Results of Open-Label Extension Study of FINTEPLA® (fenfluramine) Oral Solution in Patients with Lennox-Gastaut Syndrome (LGS)

Retrieved on: 
Wednesday, November 23, 2022
LGS, OLE, Patient, Children's Hospital Colorado, Fenfluramine, Communication, Death, Sleep, Seizure, UCB, GTCS, Valvular heart disease, FAES, Hypertensive emergency, Children's Hospital, MSCS, Associate professor, Generalized tonic–clonic seizure, Safety, Pharmaceutical industry, Euronext, Epilepsy

In that randomized trial, a total of 263 patients were assigned to receive either FINTEPLA 0.7 mg/kg/day (n=87) or FINTEPLA 0.2 mg/kg/day (n=89) or placebo (n=87).

Key Points: 
  • In that randomized trial, a total of 263 patients were assigned to receive either FINTEPLA 0.7 mg/kg/day (n=87) or FINTEPLA 0.2 mg/kg/day (n=89) or placebo (n=87).
  • A total of 247 eligible patients with LGS who completed that 14-week randomized Phase 3 clinical trial enrolled in the OLE study.
  • FINTEPLA is indicated for the treatment of seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in patients 2 years of age and older.
  • If FINTEPLA is coadministered with strong CYP1A2 or CYP2D6 inhibitors, the maximum daily dosage of FINTEPLA is 20 mg.