Another candidate for the upcoming blockbuster club of Novartis is here.
The Swiss drug manufacturer received FDA approval on Friday for Adakveo, the first targeted therapy to prevent painful vaso-occlusive crises (VOCs) associated with sickle cell disease. The nod came weeks ahead of schedule, even after a priority review in July.
Novartis has evaluated the new drug, which is intended for patients over the age of 16, for a wholesale price of $ 2,357 per vial. Because of the weight, most patients would take three or four vials per month. This would mean a WAC of $ 7,071 or $ 9,428 per month.
In Adakveo, Novartis seeks to close an innovation gap in sickle cell disease and reach a billion-dollar peak Sales; The drug is one of a dozen potential blockbuster launches announced by CEO Vas Narasimhan until 2021.
"The biggest contributing factor patients complain about today, despite all available care standards, is VOC," said Andrew Cavey, Novartis Global Program The head of the Department of Benign Hematology announced FiercePharma prior to approval. "And these are unpredictable, serious events that can cause life-threatening complications."
Every VOC event affects a patient's quality of life, causes new organ damage, and increases the risk of death.
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Adakveo aims to reduce VOC by inhibiting P-selectin protein, which plays a key role in how various cells in blood vessels adhere to one another and cause VOC. Novartis acquired the drug in 2016 through the purchase of Selexys Pharmaceuticals for $ 665 million.
In a Phase 2 study entitled Sustain, Adakveo was able to demonstrate that the average annual VOC rate was reduced by 45% or 1.63 incidents compared to 2.98 compared to placebo. In the 52-week study, up to 36% of patients on Adakveo had no VOC at all, compared with 17% in the placebo arm. It also reduced the median of days on which a patient was hospitalized in one year by 42%.
This last improvement is not only relevant to patients, but could also position Adakveo well in reimbursement discussions with payers, as it actually lowers health care costs, Cavey said.  At launch, Cavey said Novartis could leverage his experience in hematology, pointing out that about half of his oncology portfolio is blood-borne.
Indeed, Novartis already has relationships with patients with sickle cell disease and their physicians on Exjade / Jadenu, which is approved for the treatment of high iron levels due to blood transfusions, he noted. Patients with sickle cell disease may receive a blood transfusion to relieve the symptoms.
RELATED: GlycoMimetics Shares Almost Halve as Phase 3 Drug Flops with Pfizer Partners Endari is an oral powder formulation of the amino acid L-glutamate that functions as hemoglobin, the oxygen transporter of the blood , should improve. Approved by the FDA in 2017 as the first treatment for sickle cell disease in about two decades, the drug failed to materialize in the first nine months of 2019 and generated only $ 17.3 million in revenue.
Rivipansel from GlycoMimetics, partner of Pfizer, which also aims to block the activity of selectin cell adhesion molecules, has just skipped phase 3 in patients with sickle cell disease in the hospital in the middle of a VOC. The primary endpoint of discharge readiness and some important secondary endpoints has failed across the board.
There is also the potential threat of gene therapy. Although it may take years, Bluebird Bio has begun to clinically test the EU-approved beta thalassemia gene therapy Zynteglo (LentiGlobin) for severe sickle cell disease.
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Novartis, known for its interest in novel gene and cell therapies, has invested in this field in collaboration with Intellia Therapeutics. The pair uses Intellia's CRISPR-Cas9 gene editing platform to optimize human hematopoietic stem cells.
By increasing the production of fetal hemoglobin to replace crescent red blood cells, the approach promises to alleviate the disease. Companies reported encouraging ex vivo results by the end of 2017, and Cavey announced that they hope to be admitted to the clinic soon.