Leukodystrophies

Autobahn Therapeutics Announces Orphan Drug Designation Granted to ABX-002 for the Treatment of X-linked Adrenoleukodystrophy

Retrieved on: 
Monday, August 2, 2021
FDA, Health, Genetics, Clinical trials, Pharmaceutical, Biotechnology, Health, Life sciences, Pharmaceuticals policy, Adrenoleukodystrophy, Leukodystrophies, Orphan drug

Autobahn Therapeutics, a biotechnology company focused on restoring hope for people affected by CNS disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the companys investigational product candidate, ABX-002, for the treatment of patients with X-linked adrenoleukodystrophy (X-ALD).

Key Points: 
  • Autobahn Therapeutics, a biotechnology company focused on restoring hope for people affected by CNS disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the companys investigational product candidate, ABX-002, for the treatment of patients with X-linked adrenoleukodystrophy (X-ALD).
  • Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States.
  • Autobahn Therapeutics is focused on restoring hope for people affected by CNS disorders.
  • Autobahn is leveraging its brain-targeting chemistry platform to unlock new therapeutic opportunities through precision tuning of the central exposure of its molecules.

SwanBio Therapeutics Announces First Patient Enrolled in Natural History Study to Evaluate Patients with Adrenomyeloneuropathy

Retrieved on: 
Tuesday, June 29, 2021
Mental health, Health, Genetics, Physical therapy, Other Health, General Health, Clinical trials, Neurological disorders, Health, Rare diseases, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy, ALD, Clinical trial, AMN, Adrenomyeloneuropathy, SwanBio Therapeutics, ADRENOMYELONEUROPATHY, SWANBIO THERAPEUTICS

CYGNET is a natural history study of adrenomyeloneuropathy (AMN), a form of adrenoleukodystrophy (ALD) occurring in adulthood.

Key Points: 
  • CYGNET is a natural history study of adrenomyeloneuropathy (AMN), a form of adrenoleukodystrophy (ALD) occurring in adulthood.
  • This observational, multinational study will prospectively evaluate patients to assess the course of the disease and provide insights into potential endpoints and designs for future clinical trials.
  • We are pleased to initiate this natural history study for AMN, which has been strategically designed with patients in mind, and allows for the majority of monitoring to be conducted virtually from each patients home.
  • The global study will enroll approximately 80 adult male patients with a confirmed diagnosis of adrenoleukodystrophy (ALD).

$833+ Million Krabbe Disease Treatment Industry to 2027 - Impact of COVID-19 on the Market

Retrieved on: 
Thursday, April 8, 2021
Rare diseases, Krabbe disease, Leukodystrophies, Krabbe, Compound annual growth rate, Branches of biology, Organ systems, Health

DUBLIN, April 8, 2021 /PRNewswire/ -- The "Krabbe Disease Treatment - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • DUBLIN, April 8, 2021 /PRNewswire/ -- The "Krabbe Disease Treatment - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.
  • Amid the COVID-19 crisis, the global market for Krabbe Disease Treatment estimated at US$833.8 Million in the year 2020, is projected to reach a revised size of US$1.2 Billion by 2027, growing at a CAGR of 5.6% over the analysis period 2020-2027.
  • The U.S. Market is Estimated at $245.8 Million, While China is Forecast to Grow at 5.2% CAGR
    The Krabbe Disease Treatment market in the U.S. is estimated at US$245.8 Million in the year 2020.
  • Led by countries such as Australia, India, and South Korea, the market in Asia-Pacific is forecast to reach US$140.3 Million by the year 2027.

Global Krabbe Disease Treatment Industry (2020 to 2027) - Market Trajectory & Analytics - ResearchAndMarkets.com

Retrieved on: 
Tuesday, April 6, 2021
Health, Pharmaceutical, Medical specialties, Clinical medicine, Medicine, Krabbe disease, Leukodystrophies, Hematopoietic stem cell transplantation, Krabbe, Compound annual growth rate

The "Krabbe Disease Treatment - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Krabbe Disease Treatment - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.
  • The U.S. Market is Estimated at $245.8 Million, While China is Forecast to Grow at 5.2% CAGR
    The Krabbe Disease Treatment market in the U.S. is estimated at US$245.8 Million in the year 2020.
  • In the global HSCT segment, USA, Canada, Japan, China and Europe will drive the 6.5% CAGR estimated for this segment.
  • Led by countries such as Australia, India, and South Korea, the market in Asia-Pacific is forecast to reach US$140.3 Million by the year 2027.

FDA Clears IND Application for Passage Bio’s Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare Pediatric Disorder with No Approved Disease-Modifying Treatment Options

Retrieved on: 
Monday, February 8, 2021
Rare diseases, Lipid storage disorders, Krabbe disease, Leukodystrophies, Organ systems, Galactosylceramidase, Health, Krabbe, Medicine

Underscoring the urgent medical need in the patient population, the FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 for treatment in Krabbe disease.

Key Points: 
  • Underscoring the urgent medical need in the patient population, the FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 for treatment in Krabbe disease.
  • PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C).
  • PBKR03 has the potential to treat both the central nervous system and peripheral nerve manifestations observed in Krabbe disease patients.
  • The trial is designed as a dose escalation study of a single ICM dose of PBKR03 in pediatric subjects with early infantile Krabbe disease.

Orchard Therapeutics Announces OTL-200 Granted Regenerative Medicine Advanced Therapy (RMAT) Designation by FDA for the Treatment of Metachromatic Leukodystrophy (MLD)

Retrieved on: 
Thursday, January 14, 2021
Branches of biology, Neurological disorders, Life sciences, Rare diseases, Lipid storage disorders, Biotechnology, Leukodystrophies, Metachromatic leukodystrophy, Gene delivery, Gene therapy, Therapy, Arylsulfatase A

BOSTON and LONDON, Jan. 14, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Jan. 14, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • This data set includes post-treatment follow-up data of up to eight years in the earliest treated patients in these programs.
  • The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies.
  • OTL-200 is an investigational therapy in the U.S.
    Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

Adrenoleukodystrophy Pipeline Review, H2 2020 Edition - Therapeutics Development and Assessment, Drug Profiles, Dormant Projects, Product Development Milestones, Competitive Landscape - ResearchAndMarkets.com

Retrieved on: 
Tuesday, January 12, 2021
Genetics, Pharmaceutical, Health, Neurological disorders, Rare diseases, Organ systems, Branches of biology, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy

Adrenoleukodystrophy - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Adrenoleukodystrophy (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.

Key Points: 
  • Adrenoleukodystrophy - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Adrenoleukodystrophy (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
  • The Adrenoleukodystrophy (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) and features dormant and discontinued projects.
  • The pipeline guide reviews pipeline therapeutics for Adrenoleukodystrophy (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • Formulate corrective measures for pipeline projects by understanding Adrenoleukodystrophy (Genetic Disorders) pipeline depth and focus of Indication therapeutics.

Global Adrenoleukodystrophy (ALD) Epidemiology Forecast to 2030 - ResearchAndMarkets.com

Retrieved on: 
Friday, December 11, 2020
Retail, Other Retail, Neurological disorders, Rare diseases, Organ systems, Branches of biology, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy, ALD, Epidemiology

The "Adrenoleukodystrophy (ALD) - Epidemiology Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Adrenoleukodystrophy (ALD) - Epidemiology Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.
  • This 'Adrenoleukodystrophy (ALD) - Epidemiology Forecast to 2030' report delivers an in-depth understanding of the disease, historical and forecasted Adrenoleukodystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
  • As per the publisher's analysis, in 2017, the total prevalent population of Adrenoleukodystrophy in the 7MM was 55,242.
  • What will be the growth opportunities in the 7MM concerning the patient population of Adrenoleukodystrophy?

Autobahn Therapeutics Completes Successful Pre-IND Meeting with FDA

Retrieved on: 
Tuesday, December 1, 2020
Biotechnology, FDA, Health, Clinical trials, Neurological disorders, Rare diseases, Organ systems, Branches of biology, Adrenoleukodystrophy, Leukodystrophies, AMN, ALD

Autobahn has gained alignment with FDA on the initial Phase 1a/b clinical trial protocol for ABX-002 as a monotherapy treatment for a rare neurological disorder, adrenomyeloneuropathy (AMN).

Key Points: 
  • Autobahn has gained alignment with FDA on the initial Phase 1a/b clinical trial protocol for ABX-002 as a monotherapy treatment for a rare neurological disorder, adrenomyeloneuropathy (AMN).
  • AMN is a progressive and debilitating form of X-linked adrenoleukodystrophy (X-ALD) with no effective treatment options.
  • Autobahn Therapeutics is focused on restoring hope for people affected by CNS disorders.
  • Autobahn is leveraging its brain-targeting chemistry platform to unlock new therapeutic opportunities by tuning the central exposure of its molecules.

NORD Launches Innovative Natural History Study on Metachromatic Leukodystrophy (MLD)

Retrieved on: 
Thursday, October 1, 2020
Health, Neurological disorders, Rare diseases, Organ systems, Leukodystrophies, Metachromatic leukodystrophy, MLD, Food and Drug Administration, Center for Biologics Evaluation and Research, Clinical trial

WASHINGTON, Oct. 1, 2020 /PRNewswire/ --Today, the National Organization for Rare Disorders (NORD) opened registration for a natural history study on metachromatic leukodystrophy (MLD), a lethal rare diseasecurrently without an FDA-approved treatment.

Key Points: 
  • WASHINGTON, Oct. 1, 2020 /PRNewswire/ --Today, the National Organization for Rare Disorders (NORD) opened registration for a natural history study on metachromatic leukodystrophy (MLD), a lethal rare diseasecurrently without an FDA-approved treatment.
  • NORD's Natural History Of MEtachromatic Leukodystrophy (HOME) Study represents an opportunity to address an area of unmet need, providing dynamic data collection and a novel framework for building regulatory-grade rare disease natural history studies incorporating patient-reported information.
  • This pilot project is funded through an award (#75F40119C10091*) to NORD from the US Food & Drug Administration (FDA), Center for Biologics Evaluation and Research (CBER).
  • The goals of the study are to enhance understanding of metachromatic leukodystrophy, inform methods for building natural history studies to serve as external controls, reduce the burden for patient participation in clinical trials, and provide innovative methods for the use of natural history study data to help accelerate therapeutic drug development and FDA decision-making.