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The information posted here does not represent an endorsement of a particular product by the SCAGO. Statements and opinions expressed here do not necessarily represent the opinions, policies, or recommendations of the SCAGO
Pfizer Canada’s Recommendations Regarding the Development of a National Strategy for Drugs for Rare Diseases
Pfizer Inc- June 7, 2022: While many countries have moved toward a Rare Disease framework to facilitate access to innovative medicine for patients with rare diseases, there is an opportunity for Pfizer Canada to play a role in defining a sustainable Rare Disease framework for Canada, working collaboratively with Health Canada, pharmaceutical industry partners, patient groups and clinicians. The following are the key questions related to our work in Canada.
What’s the situation?
Rare disease therapies face unique regulatory, health technology assessment (HTA), and reimbursement challenges. Many aspects of the current Canadian system are designed with other purposes and medicines in mind, and not taking into the unique nature of rare diseases and innovative medicines. Consequently, the availability of rare disease treatments is often delayed and inconsistent across the country.
Canadians with rare diseases encounter a wide range of challenges in managing their health. The screening, detection, and diagnosis of many rare conditions may not always be timely.
Canada continues to face inherent challenges to rare disease research. Concerted work to nurture and sustain recognized expertise, including centers of clinical excellence for rare diseases, is a key component of managing these issues.
BlueBirdBio announces the launch of its website-SparkSickleCellChange.com aimed to highlight the impact of Sickle Cell Disease on patients, their families, and the societal ecosystem through the amplification of the voices and stories of those living with Sickle Cell Disease and those caring for them.
Visit: SparkSickleCellChange.com
September 27, 2022: Vertex and CRISPR Therapeutics Announce Global exa-cel Regulatory Submissions for Sickle Cell Disease and Beta Thalassemia
BOSTON & ZUG, Switzerland–(BUSINESS WIRE)–Sep. 27, 2022– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that Vertex has concluded discussions with the U.S. Food and Drug Administration (FDA), and the FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022, and expects to complete the submission by the end of Q1 2023.
Vertex previously completed discussions with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) on the data required to support those marketing applications and is on track to submit it by the end of 2022.
“We are pleased to have concluded our exa-cel pre-submission meetings with regulators and are excited that FDA has granted a rolling review,” said Nia Tatsis Ph.D., Executive Vice President, Chief Regulatory and Quality Officer. “We continue to work with urgency to bring forward the first CRISPR therapy for a genetic disease, and one that holds the potential to transform the lives of patients with sickle cell disease or beta-thalassemia.”
Based on progress in this program to date, exa-cel has been granted multiple important regulatory designations, including Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease Designations from the FDA for both SCD and TDT. Exa-cel has also been granted Orphan Drug Designation (ODD) from the European Commission, as well as Priority Medicines (PRIME) designation from the EMA, for both SCD and TDT.
Exa-cel is being investigated in multiple ongoing clinical trials as a potential one-time therapy for patients with either SCD or TDT.