Vertex, CRISPR to begin rolling review in US for gene-edited therapy in Nov; get FDA, EMA designations

Sep. 27, 2022 8:07 AM ETVertex Pharmaceuticals Incorporated (VRTX), CRSPBy: Ravikash, SA News Editor6 Comments

Genetic engineeering concept


The U.S. Food and Drug Administration (FDA) granted a rolling review request for Vertex Pharmaceuticals (NASDAQ:VRTX) and CRISPR Therapeutics (NASDAQ:CRSP) gene-edited therapy exagamglogene autotemcel (exa-cel) to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review starting in November and expects to complete the filing by the end of Q1 2023, the companies said in a Sept. 27 press release.

Vertex added that it previously completed discussions with the European Medicines Agency (EMA) and U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) on the data required to support regulatory filings for approval and is on track to submit them by the end of 2022.

In addition, Vertex said exa-cel has been granted regenerative medicine advanced therapy, fast track, orphan drug, and rare pediatric disease designations by the FDA for both SCD and TDT.

Exa-cel has also been granted orphan drug designation and priority medicines (PRIME) designation in the EU for SCD and TDT.

The companies noted that exa-cel is being investigated in multiple ongoing trials as a potential one-time therapy for patients with either SCD or TDT.

SCD is an inherited blood disorder in which there are not enough healthy red blood cells to carry oxygen in the body. These red blood cells are shaped like sickles and become rigid and sticky which can slow or block blood flow. Thalassemia is also an inherited blood disorder which causes the body to have less hemoglobin than normal.

CRSP +3.01% to $62.94 premarket Sept. 27

VRTX +0.69% to $278.70 premarket Sept. 27

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