Amgen and US WorldMeds: A Tale of Data and Two FDA Adcomm Meetings

Pictured: Boardroom overlooking a city/iStock, ism

Pictured: Boardroom overlooking a city/iStock, ism

Though data became an issue in two separate meetings, the FDA’s Oncologic Drugs Advisory Committee made a potentially precedent-setting decision by voting in favor of US WorldMeds’ neuroblastoma treatment.

Pictured: An empty meeting room with laptops at each seat/iStock, ismagilov

As FDA Commissioner Robert Califf has argued, the agency’s advisory committee system made up of nearly 50 panels of external experts is “messy” but “hard to imagine a better way of achieving our goals.” This week, the regulator got a view of just how messy the advice of outside experts can be—at least, in the case of US WorldMeds.

Despite the lack of a randomized controlled trial for US WorldMeds’ investigational drug, the FDA’s Oncologic Drugs Advisory Committee in a potentially precedent-setting decision found that the company provided adequate data to support its benefit in high-risk neuroblastoma. In a 14-6 vote, the adcomm determined that US WorldMeds provided enough evidence from a Phase II study to conclude that eflornithine hydrochloride (DFMO) boosts event-free survival in its target pediatric patient population.

While Committee Chair Christopher Lieu, director of the Gastrointestinal Medical Oncology Program at the University of Colorado, voted in favor of DFMO, he warned that the adcomm’s vote on Wednesday might lead to a “slippery slope” in terms of the degree of clinical evidence that will be required for future drug applications in similar rare and difficult-to-study indications.

If the FDA’s Oncologic Drugs Advisory Committee gave US WorldMeds some latitude, the panel was less inclined to do so with Amgen, which sought to convert the accelerated approval of its oral G12C KRAS inhibitor Lumakras to full approval.

In a 10-2 vote against the non-small cell lung cancer drug on Thursday, the adcomm found that Amgen’s progression-free survival data from a Phase III confirmatory study could not be reliably interpreted due to the high number of study dropouts, the small sample size and potentially biased behavior of the trial’s investigators. The FDA in its briefing document also raised concerns of “systemic bias” in the study.

This week wasn’t a total wash for Amgen. On Friday, the company finally completed its $27.8 billion acquisition of Horizon Therapeutics after a legal challenge by the Federal Trade Commission was resolved last month. Earlier in the week, Amgen joined AbbVie, Gilead, Merck and Novartis in forming a new 31-member industry coalition whose goal is to push back against a proposed FTC overhaul of federal antitrust guidelines and advocate for “pro-innovation” mergers and acquisitions in the sector.

A rumored acquisition in the news this week involved Mirati Therapeutics as a potential takeover target by Sanofi, according to Bloomberg. What’s intriguing about such a proposed deal is that the KRAS cancer drug battle is starting to heat up between Mirati’s Krazati and Amgen’s Lumakras.

Greg Slabodkin is the News Editor at BioSpace. You can reach him at Follow him on LinkedIn.