Sanfilippo

Orphan designation: Recombinant human heparan-N-sulfatase Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome), 12/01/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
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Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) on 12 January 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

GC Biopharma Presents Updates on its LSD Treatments at the WORLDSymposium 2024

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Wednesday, February 14, 2024
Enzyme replacement therapy, Fabry disease, ICV, EMA, Intelligence, European, Glycolipid, ERT, Sanfilippo syndrome, Hunter syndrome, Death, MPS, Prevalence, ODD, Safety, Sanfilippo, IDS, Disorder, Patient, Hanmi Pharmaceutical, Fibrosis, Clinical trial, Knowledge, Kidney disease, Disease, Urine, Heart, Alpha-galactosidase, Phase, Male, Kidney, Glycogen storage disease, Brain, Orphan, Mouse, Pharmaceutical industry

YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.

Key Points: 
  • YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.
  • WorldSymposium 2024 is an international forum for Lysosomal Diseases experts to share and exchange insights for researching better treatment of the disease.
  • In 2012, GC Biopharma succeeded in developing the world's second treatment for Hunter syndrome, "Hunterase" solely using domestic technology.
  • GC Biopharma, together with Hanmi Pharmaceutical, is developing GC1134A/HM15421, a long-acting alpha-galactosidase that can be administered subcutaneously once a month to improve patient convenience.

Denali Therapeutics Announces New Data and Expansion of Its Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapy Programs at WORLDSymposium™

Retrieved on: 
Wednesday, February 7, 2024
Traffic barrier, Sanfilippo, Genomics, Hearing, Growth, BBB, AFL, Metabolism, Feinberg School of Medicine, Spleen, Dermatan sulfate, Patient, Cerebrospinal fluid, University of North Carolina, FDA, Cognition, COMPASS, Mouse, CSF, Physician, Hunter syndrome, MPS II, Brain, Behavior, MPS, Mucopolysaccharidosis, Hunting, University, SGSH, ETV, Liver, Alfa, Glycogen storage disease, Pharmaceutical industry

Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.

Key Points: 
  • Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.
  • New two-year peripheral biomarker data demonstrate high magnitude and sustained reduction of urine heparan sulfate and dermatan sulfate, including in participants who switched from standard-of-care enzyme replacement therapy to tividenofusp alfa, suggesting enhanced peripheral activity.
  • Denali also announced that dosing has begun in the Phase 1/2 study of DNL126 for the potential treatment of MPS IIIA.
  • “Our goal is to bring effective new medicines to MPS families as soon as possible,” said Carole Ho, M.D., Chief Medical Officer of Denali.

Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)

Retrieved on: 
Tuesday, February 6, 2024
Sanfilippo, Family, Cognitive development, Research, Patient, Cerebrospinal fluid, HS, AAV, CSF, MPS, Mitt, Pharmaceutical industry

These data are from the modified intention to treat group (mITT) in the pivotal Transpher A study (N=17).

Key Points: 
  • These data are from the modified intention to treat group (mITT) in the pivotal Transpher A study (N=17).
  • 8 of 17 patients in the mITT group who reached 24 months post-treatment achieved an overall mean percent reduction from baseline of 51% (p
  • Specifically, 15 of 17 patients had both a reduction in toxic CSF HS exposure and an improvement in cognitive function.
  • Both trials are ongoing, and patients will continue to be followed for a minimum of 5 years following treatment with UX111.

Orchard Therapeutics Announces Multiple Presentations at the 20th Annual WORLDSymposium

Retrieved on: 
Friday, February 2, 2024
Abstract, Sanfilippo, Poster, Knowledge, MLD, Patient, Diagnosis, Priority review, Biologics license application, Food, Therapy, Kyowa Kirin, BLA, Flour

BOSTON and LONDON, Feb. 02, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced 10 presentations from across its neurometabolic portfolio will be featured at the 20th Annual WORLDSymposium™ taking place February 4-9, 2024, in San Diego, California.

Key Points: 
  • BOSTON and LONDON, Feb. 02, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced 10 presentations from across its neurometabolic portfolio will be featured at the 20th Annual WORLDSymposium™ taking place February 4-9, 2024, in San Diego, California.
  • Featured presentations include updated neurocognitive and biochemical results from an ongoing proof-of-concept study of investigational OTL-201 in mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome type A, with median follow-up now extending to 2.75 years (ranging from 24 to 39 months).
  • Additional data highlights include several accepted abstracts detailing newborn screening efforts to support the timely and accurate diagnosis of metachromatic leukodystrophy (MLD), as well as an encore presentation showcasing long-term results from an updated integrated analysis of 39 patients with MLD treated with investigational OTL-200, which comprises key components of the clinical data package of the Biologics License Application (BLA) currently under Priority Review by the U.S. Food and Drug Administration.
  • In addition to the data presentations outlined below, the company will also host a sponsored symposium on Monday, February 5, at 11:45 a.m., titled “Momentum, Leadership & Diagnosis: Changing the paradigm for MLD,” featuring expert speakers from different regions sharing their knowledge and experience to establish new standards and advance newborn screening through interdisciplinary collaboration.

Kyowa Kirin successfully completes acquisition of Orchard Therapeutics, a global gene therapy leader for rare diseases

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Wednesday, January 24, 2024
Patient, MHRA, Therapy, Health care, Biotechnology, Civil service commission, European Commission, Kyowa Kirin, Sanfilippo syndrome, ADS, Food, MLD, Acquisition, Acceleration, CVR, FDA, Priority review, Sanfilippo, HSC, PDUFA, Pharmaceutical industry

TOKYO and LONDON and BOSTON, Jan. 24, 2024 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.

Key Points: 
  • TOKYO and LONDON and BOSTON, Jan. 24, 2024 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.
  • The acquisition of all outstanding shares of Orchard Therapeutics by way of a Scheme of Arrangement procedure under the UK Companies Act 2006 was completed on January 24, 2024, and Orchard Therapeutics has become a wholly-owned subsidiary of Kyowa Kirin.
  • “We are truly excited about the acquisition of Orchard Therapeutics, a leading provider of HSC gene therapy.
  • Kyowa Kirin anticipates significant synergies with Orchard Therapeutics and plans to announce its 2024 Financial Guidance in conjunction with its 2023 earnings on 7 February.

Kyowa Kirin Successfully Completes Acquisition of Orchard Therapeutics, a Global Gene Therapy Leader for Rare Diseases

Retrieved on: 
Wednesday, January 24, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Patient, MHRA, Therapy, Health care, Civil service commission, European Commission, Kyowa Kirin, Sanfilippo syndrome, ADS, Food, MLD, Acquisition, Acceleration, CVR, FDA, Priority review, Sanfilippo, HSC, PDUFA, Pharmaceutical industry

Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.

Key Points: 
  • Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.
  • The acquisition of all outstanding shares of Orchard Therapeutics by way of a Scheme of Arrangement procedure under the UK Companies Act 2006 was completed on January 24, 2024, and Orchard Therapeutics has become a wholly-owned subsidiary of Kyowa Kirin.
  • “We are truly excited about the acquisition of Orchard Therapeutics, a leading provider of HSC gene therapy.
  • Kyowa Kirin anticipates significant synergies with Orchard Therapeutics and plans to announce its 2024 Financial Guidance in conjunction with its 2023 earnings on 7 February.

Denali Therapeutics Announces Key Anticipated 2024 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases

Retrieved on: 
Monday, January 8, 2024
CSF, Lysosomal storage disease, Cerebrospinal fluid, Risk, Sanfilippo syndrome, Amyloid, BBB, UC, Sanfilippo, Manuscript, ETV, ALS, OTV, SNCA, IDS, Asos, Conference, Cognition, RIPK1, Fluid mechanics, Neuroinflammation, Premedication, Glycogen storage disease, Amyloid beta, SGSH, Therapy, COMPASS, Traffic barrier, Hunting, N-sulfoglucosamine sulfohydrolase, Hunter syndrome, ISR, NewCo, Amyloid-related imaging abnormalities, Inflammation, CNS, Neurodegenerative disease, Takeda, Biomarker, Alpha-synuclein, K2, Biology, DSM-IV codes, LUMA, Brain, MPS, Investigational New Drug, PTV, ARIA, Nerve, AFL, Pathology, MPS II, Interim, BioRxiv, Plaque, MAPT, LRRK2, Elevator mechanic

The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.

Key Points: 
  • The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.
  • Upon completion of the ongoing Phase 1/2 study, and together with data from COMPASS, this combined data package is intended to support registration.
  • Increased RIPK1 activity in the CNS is hypothesized to drive neuroinflammation and cell necroptosis and to contribute to neurodegeneration.
  • Denali will maintain ownership of and continue to advance its current portfolio of clinical stage small molecule programs.

U.S. FDA Grants Orphan Drug Designation to JR-441 for the Treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA)

Retrieved on: 
Friday, December 15, 2023
FDA, Health, Science, Pharmaceutical, Research, Food, MPS, European Commission, ODD, Disease, Clinical trial, Civil service commission, Patient, BBB, JCR, NCT, TSE, ClinicalTrials.gov, Sanfilippo, Traffic barrier, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced that the U.S. Food and Drug Administration (“FDA”) granted orphan drug designation (ODD) to JR-441, an investigational drug for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome type A).

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced that the U.S. Food and Drug Administration (“FDA”) granted orphan drug designation (ODD) to JR-441, an investigational drug for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome type A).
  • JR-441 is a blood-brain barrier (BBB)-penetrating form of recombinant heparan N-sulfatase that was developed using JCR’s proprietary J-Brain Cargo® BBB-penetrating technology.
  • JCR is currently conducting a global Phase I/II clinical trial for JR-441, with the first patient with MPS IIIA dosed in October 2023.
  • We look forward to advancing JR-441 in clinical development and bringing this very important treatment to patients with MPS IIIA as quickly and safely as possible."

Denali Therapeutics Reports Third Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Tuesday, November 7, 2023
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SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the third quarter ended September 30, 2023, and provided business highlights.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the third quarter ended September 30, 2023, and provided business highlights.
  • Denali has selected five ASO targets for further development and is focused on advancing two OTV candidates towards clinical development.
  • 2023 Guidance on Operating Expenses:
    Cash, cash equivalents, and marketable securities were approximately $1.12 billion as of September 30, 2023.
  • General and administrative expenses were $25.3 million and $23.3 million for the three months ended September 30, 2023 and 2022, respectively.