Novartis P-Optin inhibitors are recommended by CHMP.

ChMP's positive comments will now be reviewed by the European Commission (EC), which usually makes a final review decision within two months.
if approved, Adakveo would be the first targeted therapy in Europe to prevent VOC in SCD patients.
VOC is sudden, unpredictable, and associated with an increased risk of organ damage and death.
clinical data show that Adakveo significantly reduced the incidence of VOC and significantly fewer days of hospitalization compared to placebo when using or not using hydroxyurea therapy (HU/HC).
Adakveo, which was the world's first in November 2019 in the United States, has been approved in the United States and seven other countries for use in Adult and Pediatric patients aged 16 and over with SCD, reducing the frequency of VOC or pain risk.
noteworthy, Adakveo is the first and only approved target biologic spent in combination with P-selectin.
in the United States, the FDA has previously granted Adakveo breakthrough drugs qualification and priority review.
P-selector is a cell-adhesive protein that plays a central role in multicellular interactions that cause vascular obstruction.
Adakveo's approval marks a new era for SCD treatment.
sickle cell disease (SCD) refers to a group of hereditary red blood cell diseases named after red blood cells in the form of "C" or "sickle".
Patients with SCD are prone to vasculatous vascular obstructive risk (VOC), especially vascular obstructive pain, which is the main reason for SCD patients seeking medical care, but the current program to prevent VOC is very limited.
VOC is triggered by clusters of cells that adhere to or block blood flow in multiple cells and are associated with increased morbidity and mortality.
by targeting P-selectors, Adakveo is effective in reducing multicellular adhesion.
chMP's positive review, based on positive data from Phase II SUSTAIN Clinical Studies.
this is a multicenter, multi-country, randomized, placebo-controlled, double-blind, 12-month study designed to assess the efficacy and safety of VOC in patients with SCD prevention in Adakveo-combination or non-hydroxyurea therapy.
results showed that Adakveo (5mg/kg) significantly reduced the median annual incidence of VOC by 45.3% (1.63 vs 2.98, p-0.010) compared to placebo sauverts.
clinically significantly significantly reduced VOC frequencies were observed regardless of SCD genotype or hydroxyurea use.
, the study showed that the proportion of patients in the Adakveo (5 mg/kg) treatment group who did not experience any VOC during treatment was more than 2 times higher than in the placebo group (36% vs 17%, p-0.010), the median of the first VOC occurred Time was 3 times that of the placebo group (4.07 months vs 1.38 months, p 0.001), and the median annual length of hospitalization decreased by 42% (4.00 days vs 6.87 days, p-0.45).
safety, the most common adverse reactions (incidence of 10%) in patients treated with 5mg/kg Adakveo (n-111) included back pain, nausea, fever and joint pain.
most adverse reactions were mild to moderate (level 1 or 2).
severe (level 3) joint pain and fever by 0.9% (1 case) each.
according to the analysis, no patients stopped treatment because of adverse reactions.
in the SUSTAIN study, there was no significant increase in adverse events of overall infection (53.0% vs 53.2%) or neutrophil reduction (3.1% vs 6.5%) reported by the Adakveo treatment group compared to the placebo group.
VOC, also known as Sickle Cell Pain Vision (SCPC), is an unpredictable and extremely painful type of event that can lead to severe acute and chronic life-threatening complications and death.
VOC can also lead to heavy use of health care, which is the most common cause of emergency room visits and hospitalizations for SCD patients, with an average lifetime medical expense of about $1 million per patient and more than $1.1 billion per year in the United States.
in SCD patients, VOC occurs when multiple blood cells stick together and stick to blood vessels, causing blockages.
reducing the stickiness of blood cells and blood vessels may help reduce the number of days patients experience VOC.
Adakveo's active drug ingredient is crizanlizumab, an anti-P-selectin monoclonal antibody that selectively binds to P-selectin on the surface of endothelial cells and on plates of blood vessels, leading to the blocking of P-selectin, inhibiting the interaction between endothelial cells, platelets, red blood cells, diseased red blood cells and white blood cells.
P-selector is one of the main drivers of vasoconcinal obstruction (VOC), a complication of SCD pain.
currently, Adakveo has been developed for the prevention of VOC in SCD patients.
SUSTAIN is part of the SENTRY Clinical Research Project, which includes a number of clinical studies aimed at obtaining comprehensive data from crizanlizumab for Clinical Management of SCD.
original source: Novartis Adakveo® receivs positive CHMP opinion for the prevention of recurrent vaso-occluscrises in patients patients with sickle cell disease original title: sickle cell disease (SCD) innovative medicine! Novartis P-Opter inhibitor Adakveo has been approved by THE EUROPEAN Union CHMP and is listed in 8 countries!