Editors' (re)View: J&J cast-off anti-hypertensive wins approval; Orchard prices MLD therapy at $4.25M

March 22, 2024
Pharma Manufacturing editors Karen Langhauser and Andrea Corona comment on the notable happenings in the pharma industry from the week of March 18

Editor’s note: Welcome to Editors' (re)View, our editors’ takes on things going on in the pharma world that deserve some extra consideration.

 J&J cast-off anti-hypertensive wins approval

This week, the FDA approved Idorsia's oral anti-hypertensive therapy, Tryvio, making it the first and only endothelin receptor antagonist for the treatment of high blood pressure not adequately controlled, in combination with other antihypertensives.

What was interesting to me about this story is that back in September, with this approval clearly in site, J&J’s Janssen Biotech had bailed on its years-long $230 million collaboration with Idorsia to develop the drug.

The trial results, shared in 2022, had been positive. In the phase 3 PRECISION trial, aprocitentan significantly reduced blood pressure when added to standardized combination background antihypertensive therapy in patients with resistant hypertension.

The only negative shared in the study results had been mild-to-moderate fluid retention in 30% of patient. But only two of these adverse events were serious and only seven patients discontinued treatment due to edema.

Following those results, and given the urgent need for new therapies for people with hypertension that couldn’t be controlled using current drugs, analysts had pegged aprocitentan to be a $2.5 billion blockbuster.

When J&J bailed in September, the drug has a PDUFA date of December 19, 2023 — although it did seem as though the partners knew an impending REMS (because of the risk of embryo-fetal toxicity) was going to delay approval for three months, pushing the date into March 2024.

Even with the delay, there is no stated reason why J&J abandoned the drug. Strategic decision? Or just a misstep? We may never know. But it seems to be good news for Idorsia, who recently underwent a round of layoffs to cut costs as it awaited Tryvio’s approval. And no matter who brings it to market, Tryvio is a step forward for the millions of Americans whose blood pressure is not well-controlled despite existing therapies. —Karen Langhauser

Orchard snags FDA approval for MLD drug, prices at $4.25M

Earlier this week, the FDA approved Orchard Therapeutics' gene therapy for metachromatic leukodystrophy (MLD). A few days later, the drugmaker priced the therapy, branded Lenmeldy, at $4.25 million, making it the world's most expensive drug. 

The approval and pricing of Lenmeldy sheds light on a broader need within the industry to recognize and address the challenges presented by the cost of CGTs. 

The future of CGT pricing is being shaped by several models, including outcomes-based agreements where payments could be linked to the health outcomes of therapies, ensuring investments are made in treatments that offer real benefits to patients. Subscription-like models are being discussed, where payers would cover the costs of therapies for a specified population over a period, making expensive treatments more manageable for health care systems.

Conversations around other financing mechanisms, such as medical loans or annuity payments are also taking place, aiming to make the therapies more accessible by spreading their high costs over time.

At the same time, the U.S. government's role is changing, with an increased focus on making these therapies accessible through regulatory frameworks that encourage CGT development, while implementing pricing regulations and reimbursement strategies that balance the need for innovation with ensuring patient access to critical treatments.

As more CGTs continue to be approved, the importance of developing comprehensive, fair, and sustainable pricing and access strategies takes center stage. These efforts will define the commercial success of these therapies and more importantly their impact on public health. — Andrea Corona