Updates

Orphazyme announces withdrawal of European Marketing Authorisation Application for arimoclomol for the treatment of Niemann-Pick disease type C

March 22, 2022 06:54 ET | Source: ORPHAZYME A/S Orphazyme A/S Company announcement No. 13/2022 Inside information www.orphazyme.com Company Registration No. 32266355 Decision to withdraw the European Marketing Authorisation Application comes ahead of scheduled final vote on the application later this month Orphazyme intends to request Type C Meeting with...

The Global Community supports INPDA’s letter to EMA

The International Niemann-Pick Disease Alliance (INPDA) has organised a letter (see below) voicing the perspective and concerns of the NPC community, which will be sent to the European Medicines Agency (EMA) next week. Families also have the opportunity to...

Orphazyme Announces Update on Regulatory Review of Arimoclomol in the European Union

Orphazyme A/S Company announcement No. 07/2022 Inside information www.orphazyme.com Company Registration No. 32266355 Copenhagen, Denmark, February 23, 2022 – Orphazyme A/S (ORPHA.CO (DK); ORPH (US)) (the “Company”), a late-stage biopharmaceutical company, today announced an update on the ongoing review of the Marketing Authorisation Application (MAA)...

IB1001-301: Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study

We are pleased to share an update from IntraBio, regarding the clinical development of their lead drug, N-acetyl-L-leucine (IB1001) for NPC.  IntraBio is conducting a pivotal Phase III, randomized, double-blind, placebo-controlled trial for patients aged 4 years and older...

Orphazyme provides update for planned NDA resubmission for arimoclomol for the treatment of Niemann-Pick disease type C in the United States

Orphazyme A/S Investor news No. 02/2022 www.orphazyme.com Company Registration No. 32266355 Orphazyme has made progress towards resubmission of the NDA for arimoclomol to the FDA and plans to request a Type C Meeting in Q2 2022 Subject to these discussions, the Company aims...

Cyclo Therapeutics Announces Formation of Global Steering Committee Comprised of Leading Experts to Advise on the Global Phase 3 Clinical Development Program for Trappsol® Cyclo™ in Niemann-Pick Disease Type C

Changes to the Executive Management of Orphazyme – Anders Vadsholt to be appointed CEO

Copenhagen, Denmark, January 31, 2022 – Orphazyme A/S (ORPHA.CO; ORPH) (“Orphazyme” or the “Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces that Chief Executive Officer (CEO) Christophe Bourdon...

Cyclo Therapeutics Receives IND Clearance from the U.S. FDA to Advance its Phase 2 Study of Trappsol® Cyclo™ for the Treatment of Alzheimer’s Disease

Dec 6, 2021 GAINESVILLE, Fla. – Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with diseases, today announced it...

EMA accepts regulatory submission for olipudase alfa, the first potential therapy for ASMD

EMA accepts regulatory submission for olipudase alfa, the first potential therapy for ASMD Olipudase alfa has been granted PRIority MEdicines (PRIME) designation in Europe, Breakthrough Therapy designation in the United States, and SAKIGAKE designation in Japan European regulatory decision anticipated second half of 2022 DECEMBER 6, 2021...

Orphazyme provides regulatory update following Type A meeting with FDA on arimoclomol in Niemann-Pick disease type C

Progress made in understanding potential resolution of topics outlined in Complete Response Letter, including need for additional data to support NDA FDA recommends the company provides supplemental information and analyses, and the FDA offers to have further interactions to...