Nov 27 Sickle cell disease treatment

Novel treatment approval promises a better future for Sickle cell disease patients

Sickle cell disease is a haematological, genetic disorder that affects millions of people worldwide.

According to the NIH, “Sickle cell disease is the most common inherited blood disorder in the United States, affecting 70,000 to 80,000 Americans’’. The estimates are somewhat similar to that of CDC’s according to which SCD afflicts approximately 100,000 Americans.

Sickle cell disease preponderance is more or less equal in both the genders.

Interestingly enough, Japan has almost nil prevalence of sickle cell disease.

The disease affects 1 in 500 African Americans and 1 in 1,000 to 1,400 Hispanic Americans.

Sickle cell disease is a result of a single genetic mutation. Characterized by the abnormality in haemoglobin sickle (HbsS), it results in the formation of haemoglobin β, i.e. polymerization. It is inherited in autosomal recessive form and occurs due to homozygosity for HbS (HbSS).

Oxbryta

Sickle cell disease treatment approaches chiefly help in easing the symptoms and are curative in nature. However, recent approvals of therapies like that of Global Blood Therapeutics (GBT), are focusing on treating the disease from its root.

Oxbryta (Voxelotor), has recently received a green signal from the US FDA, is a tablet taken orally once-a-day. It blocks the polymerization of the mutant haemoglobin thus increasing its affinity to oxygen. The new therapy, as revealed by the company, will cost USD 125,000 annually to the Sickle cell disease patients.

Other pharma companies across the globe, like GBT Therapeutics, Pfizer, AstraZeneca, Ironwood Pharmaceuticals, Imara Therapeutics, and many others, are involved in developing therapies for Sickle Cell Disease.

Launch of emerging therapies, such as Rivipansel (Pfizer), IMR-687 (Imara Therapeutics), Ticagrelor (AstraZeneca), and other compelling treatments, are expected to further increase the Sickle Cell Disease market in upcoming years.

Other novel gene-edited therapies are also under development for permanent cure of Sickle cell disease. For instance, Vertex Pharma’s CTX001, an investigational ex vivo CRISPR gene-edited therapy is currently under Phase I/II stage of development in collaboration with CRISPR Therapeutics. The candidate has already received FDA Fast Track Designation early this year.

Novel and innovative therapeutic approach for treating Sickle Cell Disease, including new medications, advances in transplantation, and gene therapies are being explored, which are expected to drive the Sickle cell disease treatment market during the forecast period significantly.

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