New drugs for rare diseases! AstraZeneca C5 complement inhibitor Soliris (Suliri, ® eculizumab) is launched in China!

Nov.
12, 2022 /Biovalley BIOON/ -- AstraZeneca today announced that its rare disease group Alexion has launched its first rare disease therapy in China: Soliris (trade name: eculizumab, eculizumab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS)
in adults and children in China 。 This marks a significant expansion
of AstraZeneca's global footprint in rare diseases following the acquisition of Alexion in 2021.

In addition to announcing Soliris, AstraZeneca announced that China's National Medical Products Administration (NMPA) has accepted Soliris' supplementary application for the treatment of refractory systemic myasthenia gravis (gMG) adult patients
with positive anti-acetylcholine receptor (AchR) antibodies.
The NMPA also approved AstraZeneca's ongoing global clinical trial in China to evaluate investigational therapies for the treatment of lupus nephritis (LN), immunoglobulin A nephropathy (IgAN), immunoglobulin light chain (AL) amyloidosis
.

Soliris (eculizumab) is the world's first approved C5 complement inhibitor that works
by selectively inhibiting activation of the terminal complement C5 protein.
Currently, Soliris is approved for the treatment of PNH, aHUS, AchR antibody-positive gMG adult patients in the US, EU and Japan and anti-aquaporin-4 (AQP4) anti-antibody positive neuromyelitis optica spectrum disorder (NMOSD
).

In September 2021, AstraZeneca established a rare disease business unit
in China.
In the future, the company will introduce more innovative drugs to China
.
The launch of Solirs in China will provide a new treatment option for patients with PNH and aHUS, reducing disease symptoms and preventing further damage
to the dysregulated complement system.

Soliris' mechanism of action (image from the literature - PMID: 25753400)

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, life-threatening rare hematologic disorder characterized by complement-mediated intravascular hemolysis, potential hematopoietic failure, and a tendency to thrombosis, resulting in organ damage and even death
.
The global prevalence of PNH is about 16-20/1 million, and the current incidence in China is estimated to be about
1/100,000.
The disease tends to occur in young adults, with 77% of patients with PNH occurring
between the ages of 20 and 40.
Without effective treatment, patients with PNH have poor quality of life and short survival, with a 5-year mortality rate of 35%.

Atypical hemolytic uremic syndrome (aHUS) is a complement-mediated thrombotic microangiopathy (CM-TMA)
caused by an overreaction of the complementate system to attack healthy cells.
Patients with this extremely rare disease can form blood clots in small blood vessels throughout the body, causing sudden, life-threatening damage to
the kidneys and other vital organs.
The prevalence of aHUS is about 7 per 1 million, with a sudden onset, and the incidence in children is higher than in adults, and about 25% of children die
in the acute phase.
Nearly 50% of patients with aHUS develop end-stage renal disease (ESRD) with a mortality rate of 25%.

A 26-week randomized, double-blind, placebo-controlled trial of TRIUMPH evaluated the safety and efficacy
of Soliris in patients with PNH with hemolysis.
The findings showed that PNH patients treated with Soliris had significantly improved hemolysis and reduced
need for blood transfusion compared to placebo patients.
In addition, two pivotal studies, C08-002 and C08-003, also confirmed the safety and efficacy
of Soliris in the treatment of aHUS.
The results of the C08-002 study showed that after treatment with Soliris, platelet counts in more than 80% of aHUS patients returned to normal and were able to be maintained, and 88% of patients achieved TMA-free status and were able to maintain it; In the C08-003 study, after 26 weeks of treatment with Soliris, 80% of aHUS patients achieved TMA-free status and improved kidney function
.
To date, global data have shown Soliris to be safe and well tolerated in the long term
.
(Biovalley Bioon.
com)

AstraZeneca expands global footprint in rare disease with availability of first Alexion rare disease therapy for patients in China