Ultragenyx prices Sanfilippo gene therapy at $4M; Bristol Myers shelves Orum drug

RegulatoryapprovedSanfilippo gene therapyOrumPositive

AI Analysis

Summary

Ultragenyx received approval for its Sanfilippo gene therapy priced at $4 million, marking its second gene therapy approval since August. Bristol Myers Squibb discontinued development of its Orum drug.

Clinical Trial Data

Primary Endpoint

Met

Outcome Details

Ultragenyx's Sanfilippo gene therapy approved; Bristol Myers shelved Orum drug

Importance:7/10

Sentiment:

0.30

gene therapyFDA approvalpricingSanfilippo syndromerare diseasedrug discontinuationorphan drug voucher

Related Companies

$RARE $BMY $RHHBY

Read the original article

Published by BioPharma Dive on September 18, 2026 2:24 PM

Read Original