RVT-802

Unassigned

New Medicines

Complete DiGeorge anomaly / DiGeorge syndrome in infants

Information

New molecular entity
Enzyvant
Enzyvant

Development and Regulatory status

None
None
Not approved
Dec 20Enzyvant Sciences is a subsidiary of Sumitovant Biopharma [7].
Dec 20Has orphan drug status in EU [7].
Dec 19Enzyvant receives a Complete Response Letter to its BLA. The company intends to re-submit the BLA [7].
Jun 19Enzyvant reported that the US FDA accepted the submission of biologics license application (BLA) for RVT 802, for DiGeorge Anomaly, and the BLA received priority review, as well [6].
Jul 18Rolling submission of a Biologics License Application (BLA) initiated to US FDA.[5]
May 18Currently pre-registration according to company pipeline; assume in US [2].
May 18RVT-802 has been granted both Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy by the FDA [2].
Sep 17Enzyvant anticipates potential BLA filing in H1 2018. RVT-802 has orphan drug status in US [4].

Category

A cell-based therapy using proprietary processes to harvest, culture, and apply allogeneic thymic tissue. Transplanted into the quadriceps muscle.
Complete DiGeorge Anomaly (cDGA) is a rare disease affecting approximately 1 in 300,000 infants. Children with complete DiGeorge Anomaly are born without a thymus gland, resulting in primary immunodeficiency. The disease is almost uniformly fatal within the first 24 months of life if untreated [1].
Complete DiGeorge anomaly / DiGeorge syndrome in infants
Parenteral

Trial or other data

Dec 20PII (NCT00576836) study completed in Dec 19; PII study (NCT00576407) completed in Dec 17; and PI studies (NCT00566488 and NCT00579709) both completed in Dec 19. Only the expanded access study appears to be active [8].
Jul 20An expanded access study (NCT01220531) is continuing thymus transplantation safety and efficacy research for the treatment of complete DiGeorge anomaly. Eligible participants undergo thymus transplantation. Immune function testing is continued for one year post-transplantation. Enzyvant is a collaborator [8].
May 18PII study (NCT00576836) has completed recruitment and collection of primary outcome data; it is due to complete Jun 27 [3].
Aug 17PII study (NCT00576407) is ongoing and due to complete Jun 27 [3].
Mar 08PII study to determine whether thymus transplantation without immunosuppression is effective in treating typical complete DiGeorge syndrome completes collection of primary outcome data (NCT00576407). 26 patients will be recruited in the US. Primary outcome is survival rate at one year post-transplantation [3].
Feb 06PII study to determine whether the amount of thymus tissue transplanted into DiGeorge anomaly infants has any effect on immune outcome starts (NCT00576836). Another purpose of this study is to determine whether parental parathyroid gland transplantation (in addition to thymus transplantation) can help both the immune and the calcium problems in DiGeorge infants with hypocalcemia. 28 patients will be recruited in the US. Primary outcome is regression analyses used to correlate dose to immunologic parameters at 1 year post-transplantation: T cell proliferative response; naïve T cells; and T cell variability. Collection of these data is due to complete Aug 10 [3]