Paying for cell and gene therapy - Is the future already here?
The ascent of cell and gene therapies over the past few years has been astonishing. And their rise looks unstoppable: By 2025, the FDA expects it will be reviewing 10 to 20 of these transformative drugs per year.
But as we listen to affordability concerns from payers, providers and patients, we ’ve also had to ask, perhaps a bit provocatively, how we can afford to pay for this boom in future cures? " Without solutions to help payers manage the cost, some of our members may make the choice to exclude coverage " , stated insurer CVS Health earlier in the year in its position paper on gene therapy. “Some plans ar...
Source: EyeForPharma - August 25, 2020 Category: Pharmaceuticals Authors: Ulrich Neumann Source Type: news
After surprise rejection of hemophilia gene therapy, BioMarin asks FDA to approve dwarfism drug
A controversial drug to treat the most common form of dwarfism is heading for a federal regulator's decision.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) of San Rafael said Thursday that it submitted its new drug application for the Food and Drug Administration to approve its drug, called vosoritide, as a once-daily injection for children with achondroplasia. The bone-growth disorder prevents cartilage fr om changing into bone.
The application comes a day after the FDA rejected BioMarin's request… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - August 20, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news
After surprise rejection of hemophilia gene therapy, BioMarin asks FDA to approve dwarfism drug
A controversial drug to treat the most common form of dwarfism is heading for a federal regulator's decision.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) of San Rafael said Thursday that it submitted its new drug application for the Food and Drug Administration to approve its drug, called vosoritide, as a once-daily injection for children with achondroplasia. The bone-growth disorder prevents cartilage fr om changing into bone.
The application comes a day after the FDA rejected BioMarin's request… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - August 20, 2020 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news
FDA blocks much-anticipated BioMarin hemophilia gene therapy
BioMarin Pharmaceutical shares plunged by a third after U.S. regulators rejected its potentially game-changing gene therapy for hemophilia A patients (Source: ABC News: Health)
Source: ABC News: Health - August 19, 2020 Category: Consumer Health News Tags: Health Source Type: news
Promising gene therapy against hemophilia rejected by FDA
BioMarin had gone so far as to give its hemophilia A gene therapy a name. Now it will have to deliver more data to the FDA. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - August 19, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news
BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
SAN RAFAEL, Calif., Aug. 19, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company ' s Biologics License... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - August 19, 2020 Category: Drugs & Pharmacology Source Type: news
Include Indigenous People in COVID-19 Response
Credit: Nidwan.By Pratima GurungKATHMANDU, Jul 28 2020 (IPS) In Nepal the COVID-19 crisis has been especially hard on indigenous peoples. We had to learn a new vocabulary and use words like quarantine, self-isolation, hand sanitizers and social distancing.
We also had to respect rules that did not previously apply to our lives. Indigenous peoples are not used to washing their hands all the time because our culture is so much closer to Mother Earth and because much of the time we don’t have running water.
The situation has been even more difficult for indigenous persons with a disability, like me. I cannot keep my social...
Source: IPS Inter Press Service - Health - July 28, 2020 Category: International Medicine & Public Health Authors: Pratima Gurung Tags: Asia-Pacific Headlines Health Human Rights Indigenous Rights Source Type: news
A new cell & gene therapy approach to treat common bleeding disorder
(Wake Forest Baptist Medical Center) WFIRM researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 28, 2020 Category: International Medicine & Public Health Source Type: news
Gene Therapy Shows Promise For Hemophilia, But Could Be Most Expensive U.S. Drug Ever
(Source: NPR Health and Science)
Source: NPR Health and Science - July 20, 2020 Category: Consumer Health News Authors: Rob Stein Source Type: news
Roche, Genentech ink real-world data deal with PicnicHealth
The original focus will be on multiple sclerosis but will extend to include Huntington ' s disease and hemophilia. (Source: mobihealthnews)
Source: mobihealthnews - July 16, 2020 Category: Information Technology Source Type: news
Spark Therapeutics Announces Updated Data on SPK-8011 from Phase 1/2 Clinical Trial in Hemophilia A at ISTH 2020 Virtual Congress
Dear Investor, Please find attached a press release by Spark Therapeutics:https://sparktx.com/press_releases/isth2020/ Do not hesitate to contact us for any further questions. With best regards, (Source: Roche Investor Update)
Source: Roche Investor Update - July 13, 2020 Category: Pharmaceuticals Source Type: news
New data from phase IIIb study reinforces safety profile of Roche ’s Hemlibra in people with haemophilia A
Roche today announced results from the second interim analysis of the phase IIIb STASEY study, which reinforce the safety profile of Hemlibra ® (emicizumab) characterised in the phase III HAVEN clinical programme. (Source: Roche Media News)
Source: Roche Media News - July 13, 2020 Category: Pharmaceuticals Source Type: news
New data from phase IIIb study reinforces safety profile of Roche ’s Hemlibra in people with haemophilia A
Basel, 13 July 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from the second interim analysis of the phase IIIb STASEY study, which reinforce the safety profile of Hemlibra ® (emicizumab) characterised in the phase III HAVEN clinical programme.1,2,3 In the STASEY study, Hemlibra was effective with no new safety signals identified in adults and adolescents with haemophilia A with factor VIII inhibitors, which was consistent with previous safety observations.1 Further new interim data suggest that people on Hemlibra may be able to undergo certain minor surgeries without additional preventative (prophylac...
Source: Roche Investor Update - July 13, 2020 Category: Pharmaceuticals Source Type: news
IDELVION(R) Becomes First and Only Factor IX Therapy with 21-Day Prophylactic Dosing
European Medicines Agency Approves 21-Day Dosing Intervals for Adult Haemophilia B Patients
HATTERSHEIM, Germany, July 3, 2020 -- (Healthcare Sales & Marketing Network) -- Global biotherapeutics leader CSL Behring announced today that the European Medi... Biopharmaceuticals, Regulatory CSL Behring, IDELVION , albutrepenonacog alfa, Factor IX, haemophilia B (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 3, 2020 Category: Pharmaceuticals Source Type: news
