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Regeneron has won the first-ever FDA approval for a gene therapy to treat a rare type of inherited hearing loss caused by mutations to a key gene called otoferlin.

The treatment is the first gene therapy to be approved under the FDA Commissioner’s National Priority Voucher (CNPV) program— and the sixth treatment overall— to win approval as part of the program meant to speed drugs to market.

In parallel with the approval, Regeneron announced it has agreed to a “most favored nation” deal with the White House and the company plans to offer Otarmeni for free in the US (though that does not include the cost of administration).

It is Regeneron’s first commercial gene therapy. (Endpoints)

The FDA approval of the fixed-dose triple-combination inhaler Breztri Aerosphere positions the product as the sole US single-inhaler triple therapy for asthma in patients aged 12 years and older.

The approval marks the product’s second approved indication after its 2020 clearance for chronic obstructive pulmonary disease in adults.

Breztri combines budesonide, glycopyrrolate, and formoterol fumarate delivered via the Aerosphere pressurized metered-dose inhaler. (PharmTech)

The FDA approved Dupixent (dupilumab) for children aged 2 to 11 years with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine (H1AH) therapy — making it the first biologic authorized in the US for this age group.

The decision extends a prior approval that covered adults and adolescents 12 and older.

For the more than 14,000 US children in this age range estimated to have antihistamine-refractory CSU, it fills a treatment gap that has long left clinicians with few options beyond cycling through symptom-focused therapies. (Dermatology Times)

It also solicited public comments for a broader pilot program for real-time trials that could launch this summer. It will be aligned with an AI risk management framework developed by the National Institute of Standards and Technology.

The process could circumvent what officials call a bottleneck in drug development, in which results from trial sites are reported to manufacturers, who then analyze and submit data to the FDA.

On average, 45% of the time between a phase 1 clinical trial and submission of an application to the FDA is “dead time” spent on paperwork and other administrative tasks, he said.

The new initiative seeks to cut down on that time to move the process faster “without cutting any corners on safety,” he said.

Officials said they’ve validated signals for AstraZeneca’s trial through Paradigm Health, confirming the feasibility of real-time signal sharing.

The agency is seeking public input on questions like which clinical trial issues might benefit most from the application of AI, and if priority should be given to specific uses for AI, such as patient recruitment and safety monitoring. (Axios)

Italy’s Chiesi Group has locked in an offer to buy out KalVista Phamaceuticals for $27 per share in cash, representing a total deal value of $1.9 billion, according to an April 29 press release.

The amount floated marks the biggest transaction for Chiesi in the pharma’s nearly 100-year history. 

Chiesi is positioning the deal, expected to close in the third quarter, as a means to bolster its standing in the rare disease arena, where the company’s business unit Chiesi Global Rare Diseases already boasts substantial operations. 

The crown jewel of the transaction is Ekterly, KalVista’s oral treatment for hereditary angioedema (HAE), which bounced back from a regulatory delay to win an FDA nod last July, becoming the Massachusetts biotech’s first commercial product. (Fierce Pharma)

The freshly inked research pact focuses on creating new DNA editing tools and ultimately developing more precise treatments for genetic conditions with severe unmet need.

Profluent will use its AI models to design site-specific recombinases—enzymes that cut and rejoin DNA—for several genomic targets. Lilly will then have the chance to exclusively license and move selected assets through preclinical and clinical development, plus commercialization.

Together, the pair are putting faith in their abilities to insert larger pieces of DNA than is possible with current technology. Many genetic diseases are caused by numerous different mutations rather than a singular mutation, which can make it challenging to create targeted medicines that help all patients.

Profluent and Lilly believe they can create such therapies through a process known as kilobase-scale DNA editing. (BioSpace)

After a quarter in which sales topped $15 billion and key readouts went AstraZeneca’s way, the company is increasingly confident that its 2030 revenue target is in reach.

AstraZeneca CEO Pascal Soriot has talked up the prospect of the company hitting its $80 billion revenue target in 2030 after a quarter in which sales beat expectations and the pipeline delivered key wins.

The drugmaker reported sales of $15.3 billion in the first quarter, easing past the $14.7 billion consensus estimate of analysts that Guggenheim Securities shared in a note last month.

AstraZeneca’s 8% revenue growth on a constant exchange rate (CER) was driven by its oncology and rare disease units, which grew 16% and 15%, respectively. (BioSpace)

And it did so again in Monday’s data readout, claiming that ERAS-0015 outperformed Revolution’s drug based on a pooled analysis of two phase 1 dose-escalation trials.

Both drugs target RAS, a pathway involved in cancer that has been notoriously difficult to attack.

In press releases, it has said that ERAS-0015 has shown more binding affinity and better potency than RevMed’s asset in preclinical studies.

It’s an unusual strategy. The data from ERAS-0015 are preliminary, and cross-trial comparisons are fraught at the best of times—likely even more so with small, early-stage cancer trials.