Niemann–Pick disease

Zevra Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Updates

Retrieved on: 
Thursday, March 28, 2024
Narcolepsy, Exercise, Somnolence, New Drug Application, Patient, Visual analogue scale, Sale, NDA, G&A, Arimoclomol, Nature, IND, Bank statement, Microsoft Access, Investment, Therapy, Priority review, Corporation, Audit committee, Research, Niemann–Pick disease, Medical science liaison, Sleep disorder, Brain, Trial of the century, Safety, Interim, Food, Sleep, QuickStart, Disease, FY, Acquisition, Emetine, UCD, COL3A1, Celiprolol, Syndrome, FDA, Marketing, Conference call, Conference, EAP, SDX, NPC, Epworth Sleepiness Scale, Idiopathic hypersomnia, Physician, PDUFA, Pharmaceutical industry

CELEBRATION, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today provided corporate updates and reported its financial results for the fourth quarter and year ended December 31, 2023. 

Key Points: 
  • ET
    CELEBRATION, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today provided corporate updates and reported its financial results for the fourth quarter and year ended December 31, 2023.
  • “We made solid progress on our key priorities in 2023,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra.
  • Overview of Q4 2023 and FY 2023 Financial Results:
    Net revenue for Q4 2023 was $13.2 million, compared to prior year Q4 net revenue of $2.2 million.
  • ET, to discuss its corporate and financial results for Q4 and FY 2023.

Rafael Holdings Reports Second Quarter Fiscal 2024 Financial Results

Retrieved on: 
Wednesday, March 13, 2024
Drug development, Patient, CYTH, Niemann–Pick disease, Research, Investment, RFL

NEWARK, N.J., March 13, 2024 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL), today reported its financial results for the second quarter of fiscal 2024 – and the six months ended January 31, 2024.

Key Points: 
  • NEWARK, N.J., March 13, 2024 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL), today reported its financial results for the second quarter of fiscal 2024 – and the six months ended January 31, 2024.
  • John will be an integral member of our team, as we advance our existing assets and evaluate additional opportunities for investment,” said Bill Conkling, CEO of Rafael Holdings.
  • During the second quarter of 2024, we increased our investment in Day Three Labs and now hold a majority interest in the company with 79% of the shares outstanding and began consolidating its financial results in the second quarter of 2024.
  • During the second quarter of 2024, we increased our investment in Day Three Labs and now hold a majority interest in the company with 79% of the shares outstanding and we began reporting consolidated its financial results in January 2024.

Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients

Retrieved on: 
Tuesday, March 12, 2024
Pharmacokinetics, Niemann–Pick disease, Hospital, NPC, GM2, Patient, RAINBOW, Lead, Safety, Pharmaceutical industry

Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW ( Phase 2 RAINBOW study NCT05758922 ).

Key Points: 
  • Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW ( Phase 2 RAINBOW study NCT05758922 ).
  • The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients from the age of 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC).
  • The aim of the RAINBOW study is to determine the safety, pharmacodynamics, and pharmacokinetics of two different doses of nizubaglustat in patients, in order to identify the target dosage for Azafaros’ planned Phase 3 pivotal studies.
  • Azafaros is grateful to the patients and their families who made the decision to participate in this study.”

Zevra Therapeutics Provides FDA Update on the PDUFA Action Date for Arimoclomol as a Treatment for Niemann-Pick Disease Type C

Retrieved on: 
Monday, March 4, 2024
New Drug Application, Niemann–Pick disease, FDA, Food, PDUFA, Therapy, NPC, Patient, Disease, NDA, Pharmaceutical industry

CELEBRATION, Fla., March 04, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for arimoclomol, an investigational orally delivered, first-in-class treatment for Niemann-Pick disease type C (NPC). In addition, the FDA has set a new Prescription Drug User Fee Act (“PDUFA”) action date of September 21, 2024, and has re-affirmed its intent to present the resubmission for discussion at an advisory committee meeting to be scheduled.

Key Points: 
  • In addition, the FDA has set a new Prescription Drug User Fee Act (“PDUFA”) action date of September 21, 2024, and has re-affirmed its intent to present the resubmission for discussion at an advisory committee meeting to be scheduled.
  • As previously reported, Zevra received acceptance of the NDA resubmission for arimoclomol in early January 2024, with an original PDUFA action date of June 21, 2024.
  • The FDA has determined that the additional information constitutes a Major Amendment to the NDA, thereby resulting in an extension of the PDUFA action date.
  • As the review continues, Zevra will maintain the early access program for arimoclomol and work tirelessly to bring this potential therapy to patients as soon as possible.”

Orphan designation: Adeno-associated viral vector serotype 9 containing the human NPC1 gene Treatment of Niemann-Pick's disease, type C, 13/10/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, Notifiable disease, EMA, European Commission, Niemann–Pick disease, Patient, NPC1, Committee, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Adeno-associated viral vector serotype 9 containing the human NPC1 gene
    - Intended use
    - Treatment of Niemann-Pick's disease, type C
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2843
    - Date of designation
    - Sponsor
    FGK Representative Service GmbH
    Patients' organisations
    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.
  • 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:
    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:

Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium™

Retrieved on: 
Tuesday, January 23, 2024
RAINBOW, NPC, Glycogen storage disease, GM2, Patient, Research, GM1, Niemann–Pick disease, Gait, Science, Sleep disorder, Molecular genetics, Gangliosidosis, Safety, Central nervous system, Congress, Biomarker, Pharmacokinetics, Metabolism, Pharmaceutical industry

As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.

Key Points: 
  • As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - patients’ and caregivers’ assessments.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO)- baseline clinical data.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - evaluation of different assessments.

FDA Acceptance of Zevra’s Arimoclomol NDA Filing for Niemann-Pick Disease Type C (NPC) Results in XOMA Making a $1 Million Milestone Payment to LadRx

Retrieved on: 
Thursday, January 11, 2024
Arimoclomol, NPC, Speech, Niemann–Pick disease, Food, NDA, Cognition, FDA, New Drug Application, Pharmaceutical industry

EMERYVILLE, Calif., Jan. 11, 2024 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, announced today that based upon the U.S. Food and Drug Administration’s (FDA) acceptance of Zevra Therapeutics’ New Drug Application (NDA) for arimoclomol, an orally-delivered, first-in-class therapy for Niemann-Pick disease type C (NPC), XOMA will make a $1 million milestone payment to LadRx.

Key Points: 
  • EMERYVILLE, Calif., Jan. 11, 2024 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, announced today that based upon the U.S. Food and Drug Administration’s (FDA) acceptance of Zevra Therapeutics’ New Drug Application (NDA) for arimoclomol, an orally-delivered, first-in-class therapy for Niemann-Pick disease type C (NPC), XOMA will make a $1 million milestone payment to LadRx.
  • “NPC is an ultra-rare, progressive, neurodegenerative genetic disorder where those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills.
  • Arimoclomol has the potential to be the first approved therapy designed to slow the progress of this devastating disease,” stated Brad Sitko, Chief Investment Officer at XOMA.
  • The transaction also included a mid-single-digit to mid-teens royalty rate on commercial sales of aldoxorubicin depending upon the indication, in addition to potential payments of up to $343 million in development and commercial milestones from ImmunityBio.

Zevra Therapeutics Receives FDA Acceptance of Resubmission of NDA for Arimoclomol as a Treatment for Niemann-Pick Disease Type C

Retrieved on: 
Monday, January 8, 2024
NPC, Food, FDA, File, PDUFA, Disease, NDA, Therapy, New Drug Application, Niemann–Pick disease, Patient, Pharmaceutical industry

CELEBRATION, Fla., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has acknowledged receipt of the resubmission of the New Drug Application (NDA) for arimoclomol as an orally-delivered, first-in-class treatment for Niemann-Pick disease type C. Under the Prescription Drug User Fee Act (“PDUFA”), the FDA has deemed the arimoclomol NDA resubmission to be a Class II complete response which has a six-month review period from the date of resubmission. As a result, the FDA has assigned a PDUFA action date of June 21, 2024, and currently intends to present the resubmission for discussion in an advisory committee.

Key Points: 
  • As a result, the FDA has assigned a PDUFA action date of June 21, 2024, and currently intends to present the resubmission for discussion in an advisory committee.
  • “We are very pleased that the FDA has accepted the resubmission of the arimoclomol NDA following multiple collaborative and constructive meetings,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra.
  • “This significant milestone brings us one step closer to the potential approval of arimoclomol for a community of patients with debilitating unmet medical needs.
  • The resubmission includes additional evidence supporting trial metrics, FDA-preferred analyses, and data from multiple additional studies that provide supporting evidence of arimoclomol’s efficacy in clinical and non-clinical settings.

Human medicines European public assessment report (EPAR): Xenpozyme, olipudase alfa, Date of authorisation: 24/06/2022, Revision: 2, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Spleen, Liver, Medical Subject Headings, Marketing, Lysosome, Traffic barrier, C-reactive protein, INN, Fever, Risk, Metabolism, Niemann–Pick disease, Allergy, Caregiver, ATC, Anaphylaxis, Itch, International, Enzyme replacement therapy, Headache, Disease, Nausea, ASMD, Tissue, Brain, Blood, Language, Heart, Inflammation, Hypersensitivity, Carbon monoxide, DSM-IV codes, European Medicines Agency, CNS, Myalgia, Select, Patient, Facial muscles, Safety, Fats, Anatomy, Vomiting, IIA, Rash, Medical device

Human medicines European public assessment report (EPAR): Xenpozyme, olipudase alfa, Date of authorisation: 24/06/2022, Revision: 2, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Xenpozyme, olipudase alfa, Date of authorisation: 24/06/2022, Revision: 2, Status: Authorised

Zevra Therapeutics Announces Resubmission of Arimoclomol New Drug Application to the U.S. Food and Drug Administration

Retrieved on: 
Wednesday, December 27, 2023
NPC, Food, Caregiver, Arimoclomol, FDA, PDUFA, European, Disease, NDA, Therapy, New Drug Application, Letter, Doctor of Philosophy, Niemann–Pick disease, Patient, Safety, Pharmaceutical industry

Based on standard NDA resubmission review timelines, an acknowledgment letter from the FDA that the resubmission is complete and setting the PDUFA date is expected within 30 days.

Key Points: 
  • Based on standard NDA resubmission review timelines, an acknowledgment letter from the FDA that the resubmission is complete and setting the PDUFA date is expected within 30 days.
  • Zevra expects the NDA to be classified as a Class II submission which would be subject to a review period by the FDA within six months from the date of submission.
  • Results from this open-label trial suggest that arimoclomol reduces the long-term progression of NPC.
  • The primary efficacy trial evaluating arimoclomol for the treatment of NPC was a Phase 2/3 double-blind, placebo-controlled trial (CT-ORZY-NPC-002) of arimoclomol in 50 patients with NPC.