MeiraGTx granted RMAT designation by U.S. FDA to AAV2-hAQP1

MeiraGTx (MGTX) announced that the U.S. Food and Drug Administration, FDA, has granted Regenerative Medicine Advanced Therapy, RMAT, designation to AAV2-hAQP1 for the treatment of Grade 2/3 radiation-induced xerostomia, RIX. “This RMAT designation underscores the strength of our data indicating the potential of our AAV2-hAQP1 therapy to significantly improve the lives of patients who suffer from xerostomia following radiation treatment. The requirements for receiving an RMAT designation include that the drug candidate is an advanced regenerative medicine, in this case a gene therapy; that the therapy is targeting a serious condition, in this case, Grade 2 and Grade 3 late xerostomia caused by radiotherapy for cancers of the upper aerodigestive tract; and that the applicant has presented preliminary clinical evidence demonstrating that the drug candidate has the potential to address an unmet need in the serious condition,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx.