Mixed views on Orchid's 'arsa-cell' from ICER

US health technology assessor the Institute for Clinical and Economic Review ICER) today posted its revised  Evidence Report assessing the comparative clinical effectiveness and value of atidarsagene autotemcel (“arsa-cel”), from USA and UK-based Orchard Therapeutics (Nasdaq: ORTX), for metachromatic leukodystrophy (MLD).

“MLD is a devastating disease for children and their caregivers,” said the ICER’s chief medical officer, Dr David Rind, adding: “It is extremely rare, and most doctors have never seen a case. Parent testimony shared throughout this report has provided a window into the hope and frustration that families are feeling as new treatments become available. With arsa-cel, we see a gene therapy again filling the promise of possible treatments for conditions that previously were untreatable.”

The Evidence Report is due to be reviewed at a public meeting on September 29.