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• Following a bumpy regulatory road that involved a refusal-to-file, ImmunityBio has won FDA approval to expand Anktiva (nogapendekin alfa inbakicept-pmln) into papillary-only non-muscle-invasive bladder cancer (NMIBC).

• Wednesday’s approval comes nearly three months ahead of the FDA’s target decision date.

• The go-ahead covers Anktiva, given with Bacillus Calmette-Guérin (BCG), in BCG-unresponsive NMIBC patients with high-grade papillary-only tumors.

• The drug’s original FDA approval in 2024 is in patients with carcinoma in situ (CIS) with or without papillary tumors.

• To expand Anktiva’s label, the FDA adopted a novel approach to how it evaluates treatments for BCG-unresponsive NMIBC, the agency noted in a press release.

• The new regulatory path essentially allowed ImmunityBio to bypass an expensive randomized phase 3 trial that competitor Johnson & Johnson is currently grinding through. (Fierce Pharma)

• Patients with lesions caused by herpes simplex virus have a new FDA-approved treatment option, an oral antiviral drug from Asahi Kasei.

• The late Friday regulatory decision covers the treatment of adults whose lesions do not respond to the three oral antivirals that make up the current standard of care.

• Asahi Kasei’s once-daily pill, known in development as pritelivir, will be commercialized under the brand name Hovilpri. (MedCity News)

• Roche added a twelfth US indication to Tecentriq (atezolizumab).

• The FDA approved the programmed death-ligand 1 (PD-L1) inhibitor and its subcutaneous formulation with hyaluronidase-tqjs (Tecentriq Hybreza) for adjuvant treatment of stage III colon cancer with deficient DNA mismatch repair (dMMR), the company announced.

• The regimen pairs the immunotherapy with a fluoropyrimidine and oxaliplatin in a biomarker-defined patient group. (PharmExec)

• Ultragenyx Pharmaceutical recently received two separate FDA approvals for gene therapies in the span of a month, each regulatory nod accompanied by a voucher the company could use to speed up the future review of another rare disease drug.

• In the past week, Ultragenyx has reached separate deals to sell each regulatory fast pass.

• The Novato, California-based biotech announced Wednesday a definitive agreement to sell a voucher for $203 million, following a deal last week to sell a voucher for $210 million.

• The identities of both voucher buyers were undisclosed and the purchases still need to meet closing conditions. But the deals put Ultragenyx in line for non-dilutive capital that the rare disease specialist can use to bolster its drug pipeline.

• Congress created the voucher program to incentivize drug development in rare diseases. Companies awarded a regulatory approval in a rare pediatric disease can receive one of these vouchers, which cuts the standard review time of 10 months down to six. (MedCity News)

• Undecimal Bio launched on Tuesday with $18 million in seed financing to develop an AI-powered platform designed to identify conjugates that can deliver siRNA medicines to tissues other than the liver, a challenge that has long hampered the modality.

• The oversubscribed round was co-led by 4BIO Capital and IQ Capital. The startup, spun out of Eleven Therapeutics, plans to use the funding to expand its DELiveri platform and advance its initial programs targeting renovascular disease.

• DELiveri combines large-scale screening of synthetic compounds with AI models that analyze which chemical structures successfully deliver siRNA into cells.

• The company said it has screened over 15 million compounds across at least 10 tissues, generating more than 500 million data points on how different chemical structures perform in delivering molecules inside cells. (FirstWord)

• New details from AstraZeneca’s failed phase 3 trial of its enzyme replacement therapy for a genetic bone disease suggest the study missed its goals because of how it was composed.

• The treatment, efzimfotase alfa, is under FDA priority review with a decision due in the first half of 2027. It’s for a disease called hypophosphatasia that can cause bones to weaken, affecting strength and mobility.

• The company first reported in March that the phase 3 trial had failed after not meeting statistical significance on a test of patients’ walking. At the time, it blamed the results from a subgroup of adult patients.

• Recent data add new details. In the trial, called HICKORY, patients given injections of efzimfotase alfa for 25 weeks were able to walk an average 17.05 meters more in six minutes than they could at baseline, compared with placebo patients.

• AstraZeneca claims those results didn’t hit statistical significance in part because of a subgroup of patients with adult-onset disease. (Endpoints)

To allow for a faster and more systematic path for as many as 10,000 different rare conditions affecting 30 million Americans, the FDA said drug development should account for nuance by approaching each disease based on the evidence that can be realistically generated in small patient populations.

“We need to build on, systematize and operationalize these individual efforts into a consistently utilized, cross-center approach that champions the use of cutting-edge science, novel methodologies and innovative statistical approaches,” the FDA wrote.

• The Rare Disease Framework Initiative follows the “plausible mechanism” pathway introduced in November 2025 by former FDA Commissioner Marty Makary to expedite treatments for diseases with exceedingly small patient populations, for which a randomized trial would not be feasible.

• Positive data can validate a drugmaker’s business plan and boost its stock price, while a disappointing readout can change its course.

• These crucial findings are often presented and closely analyzed at medical conferences throughout the year — meetings where companies get to showcase the medicines they’ve staked their futures on.