Novartis eyes $1B sales with FDA nod for targeted sickle cell disease drug Adakveo

Novartis has won FDA approval for Adakveo, the first targeted therapy to prevent painful episodes of vaso-occlusive crises in sickle cell disease patients. (Novartis)

Another candidate for Novartis' future blockbuster club is here.

Friday, the Swiss drugmaker won FDA approval for Adakveo, the first targeted therapy to prevent the painful vaso-occlusive crises (VOCs) that come along with sickle cell disease. The nod came weeks ahead of schedule, even on top of a priority review doled out in July.

Novartis has priced the new drug, meant for patients 16 and older, at a wholesale acquisition cost (WAC) of $2,357 per vial. As it’s prescribed based on weight, most patients would take three or four vials a month. That would translate into WACs of $7,071, or $9,428, per month.

In Adakveo, Novartis is looking to fill an innovation void in sickle cell disease and deliver billion-dollar peak sales; the drug is among a dozen potential blockbuster launches by 2021 CEO Vas Narasimhan has laid out.

“The biggest factor today that patients complain about, despite all the available standard of care, is VOC,” Andrew Cavey, Novartis’ global program head of benign hematology, told FiercePharma ahead of the approval. “And these are unpredictable, severe events that can cause life-threatening complications.”

Each VOC event affects a patient’s quality of life, causes new organ damage and increases the risk of death, he said.

RELATED: Novartis CEO touts 25 potential—and diverse—blockbuster launches. But is its portfolio too scattered?

Adakveo is designed to reduce VOCs by inhibiting the P-selectin protein, which plays a key role in how different cells within blood vessels stick to one another and cause VOCs. Novartis acquired the drug in 2016 via its $665 million buyout of Selexys Pharmaceuticals.

In a phase 2 study dubbed Sustain, Adakveo showed it could cut the median annual rate of VOCs by 45% compared with placebo, or 1.63 events versus 2.98. As many as 36% of patients on Adakveo didn’t experience a VOC at all during the 52-week trial, compared to 17% for the placebo arm. It also reduced the median days a patient was hospitalized in a year by 42%.

That last improvement is not only relevant for patients but could also position Adakveo well in reimbursement talks with payers, because it actually lowers healthcare costs, Cavey said.

When it comes to the launch, Cavey said Novartis can leverage its experience in hematology, pointing to the fact that about half of its oncology portfolio falls in blood disorders.

In fact, Novartis already has relationships with sickle cell disease patients and their clinicians via Exjade/Jadenu, which is approved to treat high levels of iron due to blood transfusions, he noted. Sickle cell disease patients might receive blood transfusions to reduce symptoms.

RELATED: GlycoMimetics shares nearly cut in half as Pfizer-partnered phase 3 drug flops

Existing sickle cell disease therapies include Eammaus Life Sciences’ Endari, which is an oral powder formulation of amino acid L-glutamate designed to improve the function of hemoglobin, the blood’s oxygen transporter. Approved by the FDA in 2017 as the first treatment for sickle cell disease in about two decades, the drug hasn’t taken off, collecting only $17.3 million sales in the first nine months of 2019.

Pfizer-partnered GlycoMimetics’ rivipansel, which also aims to block the activity of the selectin cell adhesion molecules, just flopped a phase 3 in hospitalized sickle cell disease patients in the midst of a VOC. It failed across the board on the primary endpoint of readiness-for-discharge and some key secondary endpoints.

There’s also the potential threat from gene therapy. Even though it may be years away, bluebird bio has started clinical testing of its EU-approved beta-thalassemia gene therapy LentiGlobin in severe sickle cell disease.

RELATED: Reversing sickle cell disease with CRISPR-edited stem cells

Novartis, known for its interest in novel gene and cell therapies, has itself invested in the area through a collaboration with Intellia Therapeutics. The pair is leveraging Intellia’s CRISPR-Cas9 gene-editing platform to tweak human hematopoietic stem cells.

By increasing the production of fetal hemoglobin to replace the sickled red blood cells, the approach holds promise to ameliorate the disease. The companies reported encouraging ex vivo results in late 2017, and Cavey said they hope to enter the clinic soon.

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