# Pharmaceutical industry regulatory and corporate activity

> Live situation record from CLSTR: https://clstr.news/situations/pharmaceutical-industry-regulatory-and-corporate-activity
> Updated: 2026-09-21T14:55:32.000Z. Sources: 6. Developments: 3.

The pharmaceutical sector has experienced significant regulatory and corporate developments. The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), developed by Ultragenyx Pharmaceuticals, marking the first approved treatment for Sanfilippo syndrome Type A. This one-time gene therapy, originally developed by Abeona Therapeutics, is priced at $3.95 million in the US.

Following this approval, Andelyn Biosciences has commenced commercial manufacturing of Fayuvi at its facility in Columbus, Ohio. This marks the first FDA-approved gene therapy to be produced using the ‘Andelyn AAV Curator Platform’ process. Manufacturing is also occurring at Ultragenyx’s facility in Bedford, Massachusetts.

In addition to these developments, the industry saw corporate activity including Lisata Therapeutics’ acquisition of Marea Therapeutics and a partnership between mAbxience and Sandoz to commercialize an emicizumab biosimilar. Discussions regarding drug policy also emerged, with advocates for the Pharmaceutical Reform Alliance calling for the codification of Most-Favored-Nation pricing to manage costs.

## Timeline

### 2026-09-21: Andelyn Biosciences begins commercial manufacturing of Fayuvi gene therapy

Andelyn Biosciences has begun commercial manufacturing of Fayuvi, an FDA-approved gene therapy for Sanfilippo syndrome type A, using its AAV Curator Platform in Columbus, Ohio.

2 sources. https://clstr.news/cluster/andelyn-biosciences-begins-commercial-manufacturing-of-fayuvi-gene-therapy

### 2026-09-18: Pharmaceutical industry sees FDA approvals and major acquisitions

The FDA approved Ultragenyx’s Fayuvi for Sanfilippo syndrome, while Lisata Therapeutics acquired Marea Therapeutics and mAbxience partnered with Sandoz on a hemophilia A biosimilar.

2 sources. https://clstr.news/cluster/pharmaceutical-industry-sees-fda-approvals-and-major-acquisitions

### 2026-09-18: Ultragenyx receives FDA approval for Sanfilippo syndrome gene therapy

Ultragenyx Pharmaceuticals received FDA approval for Fayuvi, the first gene therapy for the fatal childhood disease Sanfilippo syndrome Type A, with a US list price of $3.95 million.

3 sources. https://clstr.news/cluster/ultragenyx-receives-fda-approval-for-sanfilippo-syndrome-gene-therapy

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Cite as: Pharmaceutical industry regulatory and corporate activity. CLSTR, https://clstr.news/situations/pharmaceutical-industry-regulatory-and-corporate-activity