Pharmaceutical policy

Orchard Therapeutics Announces Agreement with the Beneluxa Consortium Enabling Reimbursed Access to Libmeldy

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星期四, 一月 25, 2024
Death, Pharmaceutical policy, Patient, MHRA, Therapy, Busulfan, Health care, Civil service commission, European Commission, Health, Mutation, Food, MLD, Central nervous system, Traffic barrier, ARSA, FDA, Priority review, Genome, HSC, Nerve, PDUFA, Orchard, Pharmaceutical industry

BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “We are pleased to have reached an agreement with the Beneluxa consortium that enables sustainable access to treatment.
  • Multiple eligible MLD patients are currently in the treatment process and expected to receive Libmeldy in the coming months.

Patients in the 'Fight for Our Lives' call on Parliament to STOP the PMPRB!

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星期二, 三月 10, 2020
Health, Articles, Pharmaceutical industry, Pharmaceuticals policy, Medication, Health policy in the United States, Pharmaceutical policy

But availability to Canadian patients is seriously at risk as companies evaluate the impact of the proposed PMPRB pricing restrictions.

Key Points: 
  • But availability to Canadian patients is seriously at risk as companies evaluate the impact of the proposed PMPRB pricing restrictions.
  • The historic roles of the PMPRB are to ensure drug prices are not "excessive" and to promote research investment by the pharmaceutical industry.
  • The review board has abandoned its mandate on research investment and is extending its role on price control.
  • We call on Members of Parliament to "Stop the PMPRB and stop the harm to patients."