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The FDA granted the first product approval for the progressive kidney condition focal segmental glomerulosclerosis (FSGS), unlocking what’s believed to be a blockbuster opportunity for the drug’s developer, Travere Therapeutics.

Known as Filspari, the treatment is approved to treat adults and children at least eight years of age with FSGS but not a related condition called nephrotic syndrome. Travere estimates there to be more than 30,000 individuals in the US eligible for treatment.

Filspari has been on the market since 2023 for another kidney disease known as IgA nephropathy. There, however, Filspari faces growing competition from drugmakers advancing alternative options, threatening its revenue outlook.

Shares climbed by more than 34% in April 14 trading, pushing the company’s market value close to $4 billion. (BioPharma Dive)

A Revolution Medicines drug helped pancreatic cancer patients live nearly six months longer compared to those treated with standard of care chemotherapy, meeting the goals of a pivotal study in this aggressive malignancy with limited treatment options.

With these preliminary results in hand, Revolution said it plans to seek regulatory approval for the drug, daraxonrasib. Once the FDA accepts the submission, the agency’s review could happen quickly.

The FDA has already named Revolution’s once-daily pill as one of the medicines selected for a pilot program that cuts the review time down to one or two months for products that address national health priorities, such as cancers with few available therapies.

Revolution plans to submit the data for presentation during the upcoming ASCO annual meeting. (MedCity News)

Allogene Therapeutics’ CAR-T therapy removed all remaining detectable lymphoma cells in just over half of treated patients, according to an early look at the phase 2 data.

The Bay Area biotech is seeking to apply “off-the-shelf” cell therapy in a different way — and how its therapy fares has big implications not only for the company itself, but also for the broader allogeneic CAR-T field, which has struggled over the past few years.

While around 60% of large B cell lymphoma patients are cured by treatment with the standard combination of treatments called R-CHOP, about one-third of them relapse.

Allogene is developing its therapy to prevent disease from coming back in those patients. (Endpoints)

Amazon Web Services (AWS) is launching an artificial intelligence (AI) application to design and test novel drugs more quickly and confidently.

The system, called Amazon Bio Discovery, gives researchers direct access to a broad catalogue of specialized AI models named biological foundation models (bioFMs) that are trained on vast biological datasets.

These models evaluate and accelerate the development of new antibody therapies.

Bio Discovery clients so far include Memorial Sloan Kettering Cancer Center, Bayer and Voyager Therapeutics, and Amazon said 19 of the top 20 global pharma companies already use its AWS. (Pharmaceutical Technology)

Novo Nordisk unveiled a partnership with OpenAI, ChatGPT’s parent company, to integrate AI “globally from drug discovery to commercial operations.”

Specifically, this will involve tapping AI to analyze complex datasets, identify promising drug candidates and shorten overall R&D timelines.

Besides the planned R&D work, the partnership will involve seeking efficiencies in manufacturing, supply chain operations, and corporate functions.

Novo says the collaboration will feature “strict data protection” and human oversight to “ensure ethical and compliant use.” (Fierce Pharma)

In another signal of the deepening relationship between the pharma and AI sectors, Novartis chief executive Vas Narasimhan has been appointed to the Board of Anthropic.

Narasimhan’s appointment comes shortly after Anthropic accelerated its move into the application of AI to drug discovery by acquiring New York start-up Coefficient Bio for a reported $400 million, laying claim to a platform for target and drug discovery, as well as navigating regulatory requirements for development projects. (Pharma Phorum)

But 100% tariffs on patented pharmaceuticals and active ingredients could be devastating for some of the small and midsize biotech companies that develop more than half of all FDA‑approved medicines.

But he said tariffs are not workable for smaller companies, saying it “handicaps” them while in a biotech race with China.

“It’s very hard to take a plant in Ireland that makes one rare disease drug and tell a company, ‘You’ve got to rebuild that plant in the United States,’” he said.

The reason why might lie in a tumor-suppressing protein called p53.

Mutations in the TP53 gene that affect p53 production are among the most common drivers of cancer in humans.

People with Li-Fraumeni syndrome, who inherit a faulty gene, have a 90% chance of developing cancer by the time they turn 60, half of them before age 40.