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The FDA has given the go-ahead to Ultragenyx’s rebisufligene etisparvovec—now to be marketed under the brand name Fayuvi—for the treatment of Sanfilippo syndrome type A, an ultra-rare, progressive neurological condition in children.

Fayuvi is the first-ever FDA-approved therapy for the fatal condition, according to a company news release last week.

Sanfilippo syndrome type A causes children to gradually lose cognitive and other developmental abilities, with Ultragenyx’s new therapy indicated for patients with preserved neurodevelopmental function.

The approval represents a comeback for the one-time gene therapy, which was initially rejected in July 2025 due to manufacturing issues.

Ultragenyx expects Fayuvi will be available to ship within 30 to 60 days, with a wholesale acquisition cost of $3.95 million, Jefferies reported in a note.

That would make it one of the most expensive gene therapies of all time, trailing Orchard Therapeutics’ Lenmeldy for pediatric metachromatic leukodystrophy, which costs $4.25 million, according to a analysis.(BioSpace)

Aqneursa, known generically as N-acetyl-L-leucine, was first approved in 2024 for another rare disease called Niemann-Pick disease type C.

Ataxia-telangiectasia affects several organs and body functions, with patients confronting symptoms that include visible, dilated blood vessels and poor balance and coordination.

Aqneursa’s approval in ataxia-telangiectasia comes after the drug improved motor function compared with placebo in a Phase 3 trial.

The FDA said the treatment was the first approved for ataxia in patients with the disease. (Endpoints)

The FDA approved Lyrfigtu (lirafugratinib) for patients with previously treated bile duct cancer harboring an FGFR2 gene fusion or other rearrangement.

As Jin Yang-gon, chairman of HLB Group, Elevar’s parent company, noted in a YouTube announcement, Lyrfigtu marks the first novel cancer drug from a Korean company to secure FDA approval.

Compared with existing FGFR inhibitors such as Incyte’s Pemazyre and Taiho’s Lytgobi, Lyrfigtu was designed with increased selectivity for FGFR2, while sparing other proteins of the FGFR family, to minimize toxicities. (FiercePharma)

Merck & Co.'s Welireg (belzutifan) has secured a new kidney cancer indication, this time in the post-immunotherapy setting.

The FDA approval decision follows the drug's move into adjuvant treatment only three months ago.

Specifically, the agency today approved the oral HIF-2α inhibitor in combination with Merck and Eisai's VEGF tyrosine kinase inhibitor Lenvima (lenvatinib) for advanced renal cell carcinoma with a clear-cell component (ccRCC) following treatment with a PD-(L)1 inhibitor. (FirstWord)

FDA approved Onswik (insulin efsitora alfa-gobe), a once-weekly basal insulin indicated along with diet and exercise to control high blood sugar in adults with type 2 diabetes.

With FDA's decision, Onswik now has approval across four major regulatory regions, positioning it as a global option for patients managing type 2 diabetes.

Onswik is designed to deliver steady basal insulin levels over a full seven days, offering an alternative to the daily injections that basal insulin has traditionally required. (PharmExec)

Bayer scored another label expansion for its blockbuster-in-waiting, Kerendia. The heart and kidney med earned the first FDA nod in three decades for adults with chronic kidney disease associated with type 1 diabetes.

Kerendia (finerenone) was approved to reduce urinary albumin-to-creatine ratio (UACR), a key risk factor of worsening CKD, in patients with type 1 diabetes associated CKD.

According to Bayer, the drug stands to benefit 30% of the estimated 2 million US type 1 diabetes patients who will develop CKD within their lifetime.

UACR reduction with Kerendia is expected to reduce the risk of kidney disease progression in type 1 diabetes-associated CKD, including declining kidney filtration rates and end-stage kidney disease. (Fierce Pharma)

Overton, a New Mexico physician and White House aide, stated the MMR vaccine is safe and effective and "is our best tool right now in the public health response to the measles outbreak."

She added that "every vaccine on the U.S. market right now [is] safe and effective, according to the FDA."

She did contradict Trump's previous suggestion that Tylenol taken during pregnancy causes autism, saying she had "not seen any causal evidence."

Even though it’s been recombined in every generation through sex and gained the occasional mutation, on the whole the genome has been stable and has been passed down reliably from your ancestors.

This half is the result of genetic material that can clip itself out of the DNA sequence, float off, and re-root somewhere else.

These sequences can multiply and expand, inflating the genome from within. They can hop into the middle of another sequence and break it. They can also be fertile soil for new adaptations to grow.

Often called jumping genes for their ability to relocate in a genome, they may seem pathological — indeed, many have viral origins — or perhaps little more than junk.

But transposons are increasingly understood to be a key feature of many genetic tool kits.

Their connections to the evolution of everything from moths to wombs, and even to the fundamental biological processes that turn genes off and on, suggest that the relationship between host genome and transposon is best understood as a deep coevolutionary entanglement.