Regeneron signs drug-pricing deal, secures gene therapy approval

From STAT's O. Rose Broderick: Regeneron had a busy day yesterday.

The drugmaker struck a confidential deal with the Trump administration to reduce prices on drugs in Medicaid, the last of 17 deals initially sought by the White House last year. The company also agreed to invest $27 billion in drug development in the United States. Administration officials promised more drug-pricing agreements in the near future, specifically targeting smaller companies.

Regeneron also announced the FDA approval of Otarmeni, the first gene therapy to be green-lit under the agency's new National Priority Voucher pilot program. In early trials, the drug provided modest hearing gains for people with a rare type of hearing loss, though its development has received pushback from parts of the Deaf community. Regeneron plans to offer the drug at no cost to American patients.

Read more from STAT’s Daniel Payne and myself.

devices

Medicare tries to speed device coverage, cautiously

CMS is rolling out yet another attempt to fix the long-standing lag between FDA approval and actual reimbursement for medical devices, STAT’s Mario Aguilar and Katie Palmer write. It’s proposed a “RAPID” pathway that aligns evidence requirements earlier, so breakthrough devices could get covered faster — at least in theory.

The agencies are trying to make the process more stable and predictable: Companies work with CMS during the FDA process to design trials that satisfy both agencies. If the device companies hit agreed-upon endpoints, they get smoother coverage decisions. It’s a more disciplined version of past efforts that handed out coverage too loosely, but it stops well short of the automatic reimbursement industry has been lobbying for.

“We’re going to demystify the process by providing a clear signal to device manufacturers of exactly what targets they need to hit to get Medicare coverage,” said Medicare Deputy Administrator John Brooks.

podcast

A CAR-T biotech's dramatic turnaround, and drugmakers' tactics to drive more scripts

How did a biotech company that almost ran out of money three times get acquired for over $3 billion? Will the M&A streak continue? And why are drugmakers working with a telehealth company called Prescribery?

We discuss all that and more on this week's episode of "The Readout LOUD," STAT's weekly biotech podcast. Venture capitalist Bryan Roberts joins us to discuss his firm’s investment in Kelonia Therapeutics, the CAR-T biotech that Eli Lilly just said it would buy for $3.25 billion.

We also bring on our colleague Katie Palmer to talk through her story about pharma companies using bargain-basement telehealth providers to drive drug prescriptions and sales.

Listen here.

drug pricing

States double down on drug price boards

State lawmakers are pushing ahead with prescription drug affordability boards, despite efforts to restrict their authority. In Virginia, legislators unanimously rejected an attempt to weaken a new board that would peg drug price caps to Medicare-negotiated rates. And in Colorado, STAT’s Ed Silverman writes, a bill to exempt orphan drugs was scuttled quietly, preserving the prescription board’s authority to scrutinize a broad swath of medicines.

Although Medicare now has some negotiating power of prices, states are impatient with the slow, narrow reach of federal reforms. But the biopharma industry continues to argue these boards miss the real bogeyman when it comes to higher drug prices — pharmacy benefit managers.

regulation

FDA rejects Grace stroke drug over manufacturing issues

The FDA rejected GTx-104, an experimental treatment made by Grace Therapeutics for aneurysmal subarachnoid hemorrhage, a rare, often fatal type of stroke. The issue was not efficacy, but rather with the chemistry, manufacturing, conrols, and some non-clinical data gaps. The verdict sent Grace shares down sharply, although the door’s still open for resubmission.

GTx-104 is essentially an IV formulation of nimodipine — the only approved drug for this condition. Grace’s formulation was designed to reduce dosing errors and drug interactions.

"Potential FDA approval of ... ‌GTx-104 ⁠for the treatment of aSAH would represent the first meaningful innovation in the standard of care for these patients in more than 40 years," Grace CEO Prashant Kohli told Reuters.