Lumacaftor/ivacaftor

Disc Medicine Expands Leadership Team with Appointment of Industry Veteran Pamela Stephenson, MPH as Chief Commercial Officer

Retrieved on: 
Monday, February 26, 2024

WATERTOWN, Mass., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced the appointment of Pamela Stephenson, MPH as the company’s Chief Commercial Officer.

Key Points: 
  • WATERTOWN, Mass., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced the appointment of Pamela Stephenson, MPH as the company’s Chief Commercial Officer.
  • “We are delighted to welcome an accomplished leader such as Pamela to Disc, where her breadth of commercial experience and acumen will be instrumental as the company continues to grow,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc.
  • Most recently, she served as the Chief Commercial Officer of Albireo, which was acquired by Ipsen, spearheading the successful launch of Bylvay for a rare cholestatic liver disease.
  • The Inducement Award was approved by Disc’s Board of Directors (the “Board”), including a majority of the independent directors serving on the Board.

European Commission Approves ORKAMBI® (lumacaftor/ivacaftor) for the Treatment of Children With Cystic Fibrosis Ages 1 to <2 Years Old

Retrieved on: 
Wednesday, July 5, 2023

Vertex Pharmaceuticals today announced that the European Commission has granted approval for the label extension of ORKAMBI® (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) ages 1 to

Key Points: 
  • Vertex Pharmaceuticals today announced that the European Commission has granted approval for the label extension of ORKAMBI® (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) ages 1 to
  • “This approval will offer some of the youngest children with cystic fibrosis the chance of improved outcomes, by treating their disease at a young age,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.
  • As a result of the long-term reimbursement agreement in the UK, children ages 1 to
  • Vertex will continue to work with reimbursement bodies across the European Union, Australia and Canada to ensure access for all eligible patients.

Vertex Receives CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 1 to <2 Years Old

Retrieved on: 
Thursday, April 27, 2023

Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of ORKAMBI® (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) ages 1 to

Key Points: 
  • Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of ORKAMBI® (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) ages 1 to
  • “Symptoms and organ damage start very early in the lives of people with cystic fibrosis.
  • Treating patients as young as possible is important, as it can potentially slow the progression of this devastating disease,” said Fosca De Iorio, Vice President, International Medical Affairs at Vertex.
  • “Today’s news provides great hope that, if approved, a treatment option will be available for the first time for about 300 young children with CF who have two copies of the F508del mutation.”
    In the European Union, ORKAMBI® (lumacaftor/ivacaftor) is already approved for the treatment of people with CF who have two copies of the F508del mutation, ages 2 years and above.

Vertex Announces U.S. FDA Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 12 to <24 months

Retrieved on: 
Friday, September 2, 2022

ORKAMBI was previously approved by the FDA for use in people with CF ages 2 years and older with two copies of the F508del mutation.

Key Points: 
  • ORKAMBI was previously approved by the FDA for use in people with CF ages 2 years and older with two copies of the F508del mutation.
  • Todays approval is another important step on our journey to reach people of all ages living with cystic fibrosis who may benefit from our medicines.
  • CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract.
  • The approval in children ages 12 to

Odyssey Therapeutics Appoints Industry Leader Jeffrey M. Leiden, M.D., Ph.D., as Board Chair

Retrieved on: 
Thursday, May 19, 2022

"As a physician scientist and industry executive, Jeff has had unparalleled success in discovering and developing innovative therapeutics and building blockbuster commercial franchises.

Key Points: 
  • "As a physician scientist and industry executive, Jeff has had unparalleled success in discovering and developing innovative therapeutics and building blockbuster commercial franchises.
  • I have great confidence that Jeff's leadership and counsel as Board Chair will be invaluable to Odyssey as we deliver on our goal of treating serious human diseases to improve patients' lives."
  • Odyssey Therapeutics is a biotechnology company focused on discovering, developing, and commercializing the next generation of immunomodulators and oncology medicines.
  • For more information, please visit www.odysseytx.com and follow Odyssey Therapeutics on Twitter ( @Odyssey_Tx ) and LinkedIn .

Eloxx Pharmaceuticals Reports Positive Topline Results from Monotherapy Arms of Phase 2 Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients

Retrieved on: 
Wednesday, November 17, 2021

WATERTOWN, Mass., Nov. 17, 2021 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced positive topline results from the monotherapy arms of its Phase 2 clinical trial of ELX-02 in Class 1 cystic fibrosis (CF) patients with at least one G542X nonsense allele mutation. ELX-02 was well tolerated and achieved a statistically significant 5.4mmol/L reduction in sweat chloride in patients at the1.5mg/kg/day dose.

Key Points: 
  • Based on the statistically significant monotherapy results observed at the 1.5mg/kg/day dose, planning for the advancement of ELX-02 into Phase 3 clinical development has started.
  • The Phase 2 clinical trial of ELX-02 was designed to evaluate safety and assess biological activity in G542X nonsense mutation Class 1 CF patients as monotherapy and in combination with ivacaftor.
  • Topline results for the intra-patient dose escalation monotherapy arms are summarized below:
    ELX-02 was generally well tolerated in the trial, with no treatment-related serious adverse events noted.
  • ELX-02 is in clinical development, focusing on cystic fibrosis (US Trial NCT04135495, EU/IL Trial NCT04126473).

Eloxx Pharmaceuticals Reports Third Quarter 2021 Financial and Operating Results and Provides Business Update

Retrieved on: 
Tuesday, November 9, 2021

We are on track to report data from the ongoing Phase 2 clinical trials for ELX-02 in the fourth quarter of 2021.

Key Points: 
  • We are on track to report data from the ongoing Phase 2 clinical trials for ELX-02 in the fourth quarter of 2021.
  • We remain on track to report data from the monotherapy arms of the ELX-02 clinical trials in the fourth quarter of 2021.
  • Results for the third quarter of 2021 included costs related to the acquisition of Zikani.
  • Since the closing date of the acquisition, the results of Zikanis operations have been included in the Eloxx condensed consolidated financial statements.

Vertex Announces New Portfolio Reimbursement Agreement in Italy Including KAFTRIO®, SYMKEVI® and Additional Indications of ORKAMBI® and KALYDECO® for Eligible Patients With Cystic Fibrosis

Retrieved on: 
Friday, June 25, 2021

The agreement also expands access to KALYDECO (ivacaftor) for eligible patients ages one year and older.

Key Points: 
  • The agreement also expands access to KALYDECO (ivacaftor) for eligible patients ages one year and older.
  • In addition, the agreement covers any new approved indication extensions for Vertexs CF medicines submitted and approved for reimbursement during the term of the contract.
  • Ludovic Fenaux, Senior Vice President, Vertex International, commented, This agreement is an important milestone for cystic fibrosis patients in Italy.
  • KALYDECO was first reimbursed in Italy in 2015, followed by ORKAMBI in 2017 for patients 12 years and older.

Vertex Announces Innovative Reimbursement Agreement in Switzerland for ORKAMBI® (lumacaftor/ivacaftor) and SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for Eligible Cystic Fibrosis Patients

Retrieved on: 
Tuesday, April 21, 2020

The agreement also includes any future indication extensions for patients of different ages for SYMDEKO.

Key Points: 
  • The agreement also includes any future indication extensions for patients of different ages for SYMDEKO.
  • Commenting about todays news in Switzerland, Ludovic Fenaux, Senior Vice President, Vertex International, said, This agreement is an important milestone for the cystic fibrosis community in Switzerland.
  • Access to Orkambi and Symdeko is especially important at this time given the COVID-19 outbreak and that people with CF are vulnerable to infections.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

AllianceRx Walgreens Prime study demonstrates favorable adherence, but high cost for cystic fibrosis patients prescribed modulator medications

Retrieved on: 
Wednesday, November 6, 2019

ORLANDO, Fla., Nov. 6, 2019 /PRNewswire/ --AllianceRx Walgreens Prime and Duquesne University School of Pharmacy recently announced results from research analyzing data from cystic fibrosis (CF) patients prescribed cystic fibrosis transmembrane conductance regulator(CFTR) modulator medications.

Key Points: 
  • ORLANDO, Fla., Nov. 6, 2019 /PRNewswire/ --AllianceRx Walgreens Prime and Duquesne University School of Pharmacy recently announced results from research analyzing data from cystic fibrosis (CF) patients prescribed cystic fibrosis transmembrane conductance regulator(CFTR) modulator medications.
  • Researchers wanted to understand if patients took their medications as prescribed, as well as the financial impact of the medications on patients.
  • Since the introduction of the first CFTR modulator in 2012 and subsequent CFTR modulator approvals, these medications have significantly changed the treatment of CF.
  • Of the 3,482 patients using CFTR modulator therapies, 50.8% of patients were on lumacaftor/ivacaftor followed by tezacaftor/ivacaftor (24.8%) and ivacaftor (24.4%).