Drugs

BioMarin Natriuretic Peptide Petition Denied

FDA has denied a 6/12/2025 BioMarin petition asking for agency actions relating to C-type natriuretic peptide analogs. The denial letter says BioMarin asked that FDA:

refrain from approving any C-type natriuretic peptide (CNP) analog as a treatment for achondroplasia in any pediatric patients until 11/19/2028;
refrain from approving any CNP analog as a treatment for achondroplasia in patients less than five years of age until 10/20/2030; and
alternatively, hold an evidentiary hearing before approving any other sponsor’s CNP analog as a treatment for achondroplasia.

FDA says BioMarin’s Voxzogo (voxsoritide) has orphan drug exclusivity for the two indications and dates cited in the company’s petition. The petition, the agency says, focuses specifically on the CNP analog navepegritide for which Ascendis Pharma submitted a 505(b)(1) NDA.

“FDA declines to categorically refrain from approving any CNP analog as a treatment for achondroplasia” for the indications and dates requested, the denial says. “FDA makes decisions regarding whether an earlier-in-time drug’s unexpired orphan drug exclusivity blocks the approval of a subsequent application on a case-by-case basis, taking into account, among other things, whether the subsequent application is for the ‘same drug.’”

FDA says it has determined that Voxzogo and navepegritide are not the same drug since they don’t contain the same active moiety, and that there is no need for an evidentiary hearing.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

Read more

Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome