Adrenoleukodystrophy

Rare Disease Day 2024 - Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy - special film launched

Retrieved on: 
Thursday, February 29, 2024
Rare disease, CALD, Central nervous system, Adrenoleukodystrophy, Committee, Leukodystrophy, Hospital, X-ALD, PPAR, Therapy, CHMP, CNS, European Medicines Agency, Patient, Disease, Pharmaceutical industry

The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.

Key Points: 
  • The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.
  • Dr. Jörg-Thomas Dierks, Chief Executive Officer of Neuraxpharm, said: "The impact this disease has on patients and their families cannot be overstated.
  • We hope that this film will raise awareness and understanding of the condition."
  • Minoryx and Neuraxpharm strongly believe that leriglitazone has a positive benefit / risk balance in patients with cALD.

Rare Disease Day 2024 - Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy - special film launched

Retrieved on: 
Thursday, February 29, 2024
Rare disease, CALD, Central nervous system, Adrenoleukodystrophy, Committee, Leukodystrophy, Hospital, X-ALD, PPAR, Therapy, CHMP, CNS, European Medicines Agency, Patient, Disease, Pharmaceutical industry

The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.

Key Points: 
  • The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.
  • Dr. Jörg-Thomas Dierks, Chief Executive Officer of Neuraxpharm, said: "The impact this disease has on patients and their families cannot be overstated.
  • We hope that this film will raise awareness and understanding of the condition."
  • Minoryx and Neuraxpharm strongly believe that leriglitazone has a positive benefit / risk balance in patients with cALD.

Poxel Reports Cash and Revenue for the Full Year 2023 and Provides Corporate Update

Retrieved on: 
Thursday, February 15, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, Other Health, Merck, Kidney, USD, POC, Adrenoleukodystrophy, Merck Serono, EPO, JPY, AMN, Sumitomo Pharma, European Patent Convention, Safety, Biomarker, EUR, ALD, ADPKD, Serono, Adenosine, Pharmacokinetics, Patent, Fatty liver disease, Patient, Diabetes, Disease, NASH, Plasma, Male, DSM-IV codes, VLCFA, Pharmaceutical industry

Thomas Kuhn, Chief Executive Officer of Poxel, stated: “We remain focused and continue to concentrate all our efforts at securing financing options, prioritizing non-dilutive solution, and partnerships.

Key Points: 
  • Thomas Kuhn, Chief Executive Officer of Poxel, stated: “We remain focused and continue to concentrate all our efforts at securing financing options, prioritizing non-dilutive solution, and partnerships.
  • We’ve also progressed our advanced discussions – in some cases on an exclusive basis – with several potential partners for each of our three proprietary products.
  • Based on this conservative assumption, Poxel expects to receive 8% royalties on TWYMEEG® net sales.
  • As of December 31, 2023, total cash and cash equivalents were EUR 2.3 million (USD 2.6 million)1, as compared to EUR 13.1 million (USD 14.0 million) as of December 31, 2022.

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 January 2024

Retrieved on: 
Sunday, February 4, 2024
Civil service commission, Pneumonia, Febrile neutropenia, Eli Lilly, Triprolidine, International, Bacteria, Schizophrenia, Myelopathy, Incidence, ALD, Cefepime, Allergic rhinitis, Nasal congestion, Neutrophil, Agency, COMP, Fever, HAP, EEIG, Headache, Static pressure, Medicine, Committee, Conditional, Spinal cord, Hospital-acquired pneumonia, PTC, Neutropenia, Public, CHMP, European, AMD, Patient, Diagnosis, Marketing, RCVS, European Commission, Brain, Disease, Infection, Inflammation, Male, PRES, PRAC, Pain, Pyelonephritis, Urinary tract infection, VAP, Ageing, EMA, Geographic atrophy, Adrenoleukodystrophy, Therapy, News, INN, Duchenne muscular dystrophy, Common, Fungal infection, Vaccine

Three new medicines recommended for approvalEMA’s human medicines committee (CHMP) recommended three medicines for approval at its January 2024 meeting.The committee recommended…, Positive recommendations on new medicines, Exblifep International non-proprietary name (INN)cefepime / enmetazobactam Marketing-authorisation applicantAdvanz Pharma LimitedTherapeutic indicationTreatment of: 1)…, Ryzneuta INNefbemalenograstim alfaMarketing-authorisation applicantEvive Biotechnology Ireland LimitedTherapeutic indicationReduction in the duration of neutropenia and…, Positive recommendations on new generic medicines, Niapelf INNpaliperidoneMarketing-authorisation applicantNeuraxpharm Pharmaceuticals S.L.Therapeutic indicationTreatment of schizophreniaMore informationNiapelf: Pending…, Negative recommendations on new medicines, Nezglyal INNleriglitazoneMarketing-authorisation applicantMinoryx Therapeutics S.L.Therapeutic indicationTreatment of cerebral progression and myelopathy in male…, Syfovre INNpegcetacoplanMarketing-authorisation applicantApellis Netherlands B.V.Therapeutic indicationTreatment of geographic atrophy (GA) secondary to age-related…, Positive recommendations on extensions of indications, Abecma INNidecabtagene vicleucel Marketing-authorisation holderBristol-Myers Squibb Pharma EEIGMore informationAbecma: Pending EC decision, Prevenar 20 (previously Apexxnar) Common namepneumococcal polysaccharide conjugate vaccine (20-valent, adsorbed)Marketing-authorisation holderPfizer Europe MA EEIGMore…, Aspaveli INNpegcetacoplanMarketing-authorisation holderSwedish Orphan Biovitrum AB (publ)More informationAspaveli: Pending EC decision, Retsevmo INNselpercatinibMarketing-authorisation holderEli Lilly Nederland B.V.More informationRetsevmo: Pending EC decision, Outcome of re-examination of recommendation on non-renewal of Conditional Marketing Authorisation, Translarna INNataluren Marketing-authorisation holderPTC Therapeutics International LimitedNewsEMA confirms recommendation for non-renewal of authorisation of Duchenne…, Outcome of referral, Pseudoephedrine Marketing-authorisation holderVarious companiesMore informationPseudoephedrine-containing medicinal products, Re-examination of conclusion of referral, Synapse Labs Pvt.

Key Points: 
  • Three new medicines recommended for approvalEMA’s human medicines committee (CHMP) recommended three medicines for approval at its January 2024 meeting.The committee recommended…, Positive recommendations on new medicines, Exblifep International non-proprietary name (INN)cefepime / enmetazobactam Marketing-authorisation applicantAdvanz Pharma LimitedTherapeutic indicationTreatment of: 1)…, Ryzneuta INNefbemalenograstim alfaMarketing-authorisation applicantEvive Biotechnology Ireland LimitedTherapeutic indicationReduction in the duration of neutropenia and…, Positive recommendations on new generic medicines, Niapelf INNpaliperidoneMarketing-authorisation applicantNeuraxpharm Pharmaceuticals S.L.Therapeutic indicationTreatment of schizophreniaMore informationNiapelf: Pending…, Negative recommendations on new medicines, Nezglyal INNleriglitazoneMarketing-authorisation applicantMinoryx Therapeutics S.L.Therapeutic indicationTreatment of cerebral progression and myelopathy in male…, Syfovre INNpegcetacoplanMarketing-authorisation applicantApellis Netherlands B.V.Therapeutic indicationTreatment of geographic atrophy (GA) secondary to age-related…, Positive recommendations on extensions of indications, Abecma INNidecabtagene vicleucel Marketing-authorisation holderBristol-Myers Squibb Pharma EEIGMore informationAbecma: Pending EC decision, Prevenar 20 (previously Apexxnar) Common namepneumococcal polysaccharide conjugate vaccine (20-valent, adsorbed)Marketing-authorisation holderPfizer Europe MA EEIGMore…, Aspaveli INNpegcetacoplanMarketing-authorisation holderSwedish Orphan Biovitrum AB (publ)More informationAspaveli: Pending EC decision, Retsevmo INNselpercatinibMarketing-authorisation holderEli Lilly Nederland B.V.More informationRetsevmo: Pending EC decision, Outcome of re-examination of recommendation on non-renewal of Conditional Marketing Authorisation, Translarna INNataluren Marketing-authorisation holderPTC Therapeutics International LimitedNewsEMA confirms recommendation for non-renewal of authorisation of Duchenne…, Outcome of referral, Pseudoephedrine Marketing-authorisation holderVarious companiesMore informationPseudoephedrine-containing medicinal products, Re-examination of conclusion of referral, Synapse Labs Pvt.
  • Ltd. Marketing-authorisation holderVarious companiesMore informationSynapse

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Retrieved on: 
Sunday, February 4, 2024
Hearing, Inflammation, Civil service commission, Knowledge, European, Cell, Haematopoiesis, COMP, IRIS, Bone marrow, Mitochondrion, Adrenal insufficiency, Regulation, Gland, ABCD1, Patient, Safety, Diagnosis, EMA, ALD, European Commission, Adrenoleukodystrophy, Seizure, B cell, PPAR, Brain, Disease, Therapy, Eurostat, Tissue, Dementia, Medicine, Committee, Hormone, DSM-IV codes, Spinal cord, Pharmaceutical industry

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Key Points: 


Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Veteran Mom Says Marine Son Was Miracle Child

Retrieved on: 
Tuesday, December 5, 2023
Fisher House, Genetic testing, Meal, Travel, Adrenoleukodystrophy, Army, Hospital, Brain, ALD, Hatboro, Pennsylvania, Behavior, Transport, Diagnosis, DSM-IV codes, USS Harry S. Truman

Rockville, MD, Dec. 05, 2023 (GLOBE NEWSWIRE) -- If you talk to Army veteran Pat Sherrod-Elliott about her son, Marine Sgt.

Key Points: 
  • Rockville, MD, Dec. 05, 2023 (GLOBE NEWSWIRE) -- If you talk to Army veteran Pat Sherrod-Elliott about her son, Marine Sgt.
  • It took months at hospitals in Charleston, South Carolina and Bethesda, Maryland to get a diagnosis.
  • People born with ALD usually do not make it through childhood, which is what made Sean’s case a miracle.
  • “He loved God, loved his country, loved being a Marine, and he was a beloved father.

Poxel Provides Corporate Update and Reports Cash and Revenue for the Third Quarter and Nine Months 2023

Retrieved on: 
Wednesday, November 8, 2023
Biotechnology, Pharmaceutical, Health, Adrenoleukodystrophy, Patient, Serono, ALD, Plasma, AMN, Patent, Biomarker, Sumitomo Pharma, Diabetes, Merck, SGLT2, Jaguar 3.4 Litre, EPO, Pharmacokinetics, Male, DPP4, Disease, Prescription, Marshal of France, Pioglitazone, European Patent Office, EUR, United States patent law, Merck Serono, VLCFA, Safety, European Patent Convention, JPY, Estonia, Kidney, POC, Pharmaceutical industry, Imeglimin

Sumitomo Pharma has worked diligently to increase the production and secure inventory capacity to meet this increasing demand.

Key Points: 
  • Sumitomo Pharma has worked diligently to increase the production and secure inventory capacity to meet this increasing demand.
  • For the Sumitomo Pharma FY 2023, as a conservative assumption in line with Sumitomo Pharma’s forecast, Poxel expects to receive 8% royalties on TWYMEEG net sales.
  • On July 5th, Poxel was chosen as the winner of the 2023 edition of the I-nov contest for its program in ALD.
  • Next Financial Press Release: Fourth Quarter 2023 Cash and Revenue update, on February 15, 2024

SwanBio Advances to Higher-Dose Cohort in First-in-Human Study of Gene Therapy for Adrenomyeloneuropathy

Retrieved on: 
Wednesday, October 4, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, AMN, DSMB, Adrenoleukodystrophy, Clinical trial, Mutation, CTA, AAV, Trial of the century, Data monitoring committee, Degenerative disease, ABCD1, Patient, Central committee, Therapy, PROPEL, Safety, Pharmaceutical industry

SwanBio Therapeutics , a gene therapy company developing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced it has begun the second dose-escalation cohort of its first interventional clinical study, PROPEL.

Key Points: 
  • SwanBio Therapeutics , a gene therapy company developing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced it has begun the second dose-escalation cohort of its first interventional clinical study, PROPEL.
  • PROPEL is a Phase 1/2 first-in-human clinical trial assessing the safety and efficacy of SBT101, an investigational gene therapy designed to compensate for the ABCD1 mutation that causes adrenomyeloneuropathy (AMN).
  • An independent Data Safety Monitoring Board (DSMB) recommended progressing the study to the higher-dose cohort following the review of the initial safety data readout in August.
  • No serious adverse events related to the procedure, nor the gene therapy product, have been reported to date.

Poxel Announces its Participation at Upcoming Scientific Conference

Retrieved on: 
Monday, October 2, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, NASH, Adrenoleukodystrophy, Scientific communication, ALD, AMPK, DSM-IV codes, Adenosine, Metabolism, Polycystic kidney disease, Fatty liver disease, R, ADPKD, Pharmacology, Autosome, Pharmaceutical industry, Vaccine, Euronext

POXEL SA (Euronext: POXEL - FR0012432516) (Paris:POXEL), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced that it will participate at the upcoming 12th International Meeting on AMPK on October 4th, 2023.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516) (Paris:POXEL), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced that it will participate at the upcoming 12th International Meeting on AMPK on October 4th, 2023.
  • Sophie Bozec, Executive Vice President, R&D Pharmacology and Scientific Communication at Poxel, will host a virtual presentation on “Direct AMPK Activation: From Preclinical to Clinical.
  • Perspectives in Rare Metabolic Diseases”.
  • The presentation will review Poxel’s molecule PXL770, which is a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, focused on rare diseases treatment, including adrenoleukodystrophy (ALD) and autosomal dominant polycystic kidney disease (ADPKD).

Poxel Reports Financial Results for First Half 2023 and Provides a Corporate Update

Retrieved on: 
Tuesday, September 26, 2023
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, Diabetes, ULF, Adrenoleukodystrophy, Prescription, Plasma, JPY, Patent, Patient, Serono, Merck, R, Estonia, POC, Pioglitazone, Sumitomo Group, Disease, Male, NASH, Pharmacokinetics, Sumitomo Pharma, EPO, Fatty liver disease, Leukodystrophy, Marshal of France, Rare, Kidney, Conference, Safety, Abstract, DPP4, ALD, JDS, Merck Serono, European Patent Office, Biomarker, DSM-IV codes, AMN, IRIS, Scientific communication, United States patent law, SGLT2, VLCFA, EUR, European Patent Convention, Pharmacology, TZD, Pharmaceutical industry, Medical device, Imeglimin, Euronext

POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced its financial results for the period ended June 30, 2023 and provided a corporate update.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced its financial results for the period ended June 30, 2023 and provided a corporate update.
  • For the Sumitomo Pharma FY 2023, as a conservative assumption in line with Sumitomo Pharma’s forecast, Poxel expects to receive 8% royalties on TWYMEEG net sales.
  • On July 5th, Poxel was chosen as the winner of the 2023 edition of the I-nov contest for its program in ALD.
  • This recently issued patent provides additional protection through 2041, with the potential for an additional 5 years through patent term extension.