September 23, 2024

The need-to-know this morning

Daiichi Sankyo and AstraZeneca said the experimental cancer treatment Dato-DXd failed to prolong survival of participants in the TROPION-Breast01 clinical trial. Previously, the companies reported that the drug, an antibody-drug conjugate, achieved the study's co-primary goal of significantly delaying tumor progression.
Black Diamond Therapeutics reported preliminary mid-stage study results showing a 42% tumor response rate for its experimental drug BDTX-1535. The study participants had EGFR-mutated, non-small cell lung cancer with a further mutation known to be resistant to Tagrisso, the AstraZeneca drug that is considered standard of care.
Biohaven Pharmaceuticals said its experimental drug troriluzole achieved the primary goal of a Phase 3 study by slowing the progression of spinocerebellar ataxia, a rare neurodegenerative disease, compared to a control group of historical patient data collected between 2005-2009. The company plans to submit troriluzole for FDA approval by the end of the year. The agency previously rejected the drug in 2023.

election

A mysterious anti-pharma bus barnstorms the country

A big red bus has been touring the country lately, proclaiming in bold letters that the pharma industry is in need of reform, STAT's Rachel Cohrs Zhang reports. A new dark-money group called Americans for Pharma Reform is behind the bus, which is making the rounds in swing states in advance of the election. As to who is funding the outfit? That, for now, is a mystery.

The group is led by Rob Burgess, a Republican operative, who claims it isn't politically affiliated. Americans for Pharma Reform is calling for limits on indsutry ad spending and patent reforms, as well as for "America First" pricing.

While the group's backers are still an enigma, they could eventually be revealed in future tax filings.

PBMs

FTC files suit against the biggest PBMs

The FTC on Friday filed a lawsuit against three major PBMs — CVS Caremark, Express Scripts, and OptumRx — saying they've engaged in anticompetitive practices that have "artificially inflated" insulin prices, STAT's Ed Silverman reports. Patients with diabetes are now less able to access life-saving drugs — "thanks in part to powerful pharmacy benefit managers and their greed," Rahul Rao, the FDA's deputy director, said in a statement.

Regulators are pushing for reform, with aims to boost competition and lower drug prices. But the PBMs say they've taken steps to make insulin more accessible.

rare disease

Drug for Niemann-Pick type C approved

The FDA on Friday approved a Zevra Therapeutics' treatment for Niemann-Pick type C, a rare lysosomal storage disorder, STAT's Jason Mast writes. The approval of the drug, Miplyffa, or arimoclomol, follows years of patient advocacy and several unsuccessful drug trials. It was actually rejected in 2021 after regulators raised questions about its statistical analysis plan, causing its original developer to shutter.

But Zevra acquired the drug and resubmitted the data — and last month, an FDA advisory committee recommended its approval 11 to 5, citing both data and patient testimony suggesting the therapy could slow or stabilize the devastating neurological illness.

generic drugs

Opinion: Hatch-Waxman is in need of change

The Hatch-Waxman Act turns 40 this week, and it needs an overhaul, opines Tahir Amin, leader of a nonprofit aimed at lowering drug prices. The law helped usher generic drugs into regular use, but decades later he says it has accidentally bolstered monopolistic practices in the drug industry — which means higher drug costs. Right now, generics make up 92% of prescriptions, yet the remaining 8% account for 87% of drug spending.

Drugmakers find loopholes in Hatch-Waxman like extended exclusivity, patent thickets, and the FDA's general sluggishness in censuring companies that file improper patent listings.

Among Amin's recommendations are strengthening antitrust enforcement at the FTC, improving FDA transparency and authority, and rethinking patent linkage.

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