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Vertex's cell therapy for diabetes hits FDA roadblock

DTMT Network

The much-discussed Vertex’s novel cell therapy for the treatment of diabetes has trial has been put on hold by the United States Food and Drug Authority, the company said recently.

Vertex said that the Phase 1/2 clinical trial of VX-880 for the treatment of type 1 diabetes has been placed on clinical hold in the united states by the drug regulator as it determined that there is insufficient information to support dose escalation with the product.

Meanwhile, sharing the updates of the study, vertex said that two patients in Part A received VX-880 at half the target dose.

While the first patient dosed in Part A of the study achieved insulin independence at Day 270, with an HbA1c of 5.2%, the second patient dosed in Part A has shown robust increases in fasting and stimulated C-peptide, and reductions in exogenous insulin requirements through Day 150, the company informed.

When seen in totality, the data from the first two patients in Part A established proof-of-concept for VX-880, it added.

Expressing surprise at the hold placed on the study, Dr Carmen Bozic, Chief Medical Officer at Vertex said, “The results from the first two patients treated with half the target dose establish proof-of-concept by demonstrating that VX-880 can restore glucose-regulated insulin production and improve glycaemic control.”

Stating the achievement of insulin independence by the first patient ‘landmark milestone’ Dr Bozic added, “Further, the totality of the safety and efficacy data for all three patients dosed to date gives us high confidence in our benefit-risk assessment of VX-880 and its potentially transformative profile.”

“We are committed to working with the FDA to understand and address their questions so that the trial can resume at U.S. sites as soon as possible,” she further added.

VX-880 is an experimental allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology, is being evaluated for patients of insulin-dependent (Type1) diabetics with impaired hypoglycaemic awareness and severe hypoglycaemia and is supposed to restore the function of islet cells in the pancreas.

The drug is delivered by an infusion into the hepatic portal vein and requires chronic immunosuppressive therapy to protect the islet cells from immune rejection, Vertex informed.


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