Sickle Cell Disease Market | DelveInsight


What is Sickle Cell Disease?

Sickle Cell Disease market
Sickle Cell disease, which is also known as sickle cell anaemia. It includes a group of disorders that affect the red blood cells.Sickle Cell disease is an inherited blood disorder that is distinguished by defective haemoglobin. The changes in which red blood cells behave differently are dehydration, low oxygen levels and elevated temperature, which may cause the red blood cells to block the small blood vessels, restricting blood flow.

 
How much population is affected by Sickle Cell Disease?

Sickle cell disease is irreversible that affects males and females equally, and is responsible for increased morbidity and mortality in affected persons.
Sickle Cell Disease prevalance
There were approximately 133,385 Sickle Cell Disease prevalent cases in 2017 in the 6MM. As per DelveInsight, the United States reports for the highest Sickle Cell Disease prevalent population due to a large number of immigrants from African countries per year, followed by the EU5. Among the European countries, France has the highest Sickle Cell Disease prevalent population with around 15,000 cases followed by the United Kingdom, which has a Sickle Cell Disease prevalent population around 13,000 in 2017. Spain has the lowest Sickle Cell Disease prevalent population. Also, it was found that there is no Sickle Cell Disease prevalence in Japan since the disease does not occur in the Japanese population.

Which types of drugs are used in Sickle cell disease treatment?

Most of the Sickle Cell Disease treatments are dominated by the off label and off-patent medications (HU; Iron Chelators, Analgesics, Antibiotics, vaccines), to address the complications or for specific Sickle Cell Disease symptoms.

Hydroxyurea was the first-ever drug approved for the Sickle Cell Disease treatment in 1998. However, there is increasing evidence that Hydroxyurea is only prescribed to a fraction of patients who may be benefited from it. The most distressing issue with Hydroxyurea was the lack of FDA approval for infants with Sickle Cell Disease in the United States. However, various trials are undergoing to assess the safety and efficacy of Hydroxyurea in children. At present, it is used off-label for pediatric patients.

Sickle Cell Disease market size was majorly dependent on the use of Hydroxyurea until 2017 when Endari was launched. It was the first drug new drug for sickle cell patients in nearly two decades and was the first FDA- approved Sickle Cell Disease treatment for pediatric as well as adult patients.  

The approval of the Emmaus’s Endari for Sickle Cell Disease treatment in the United States in 2017. It had marked a significant advancement in Sickle Cell Disease treatment and is expected to pave the way for the development and approval of additional therapies.
However, the current therapies used to prevent the complications of the disease are limited, vary in their effectiveness and are associated with severe risks and tolerability issues. Besides this, the disease presents with significant unmet medical needs, with no disease-modifying Sickle Cell Disease treatment for an ongoing painful crisis.

Thus, new approaches for Sickle Cell Disease treatment, including new medications, advances in transplantation, and gene therapies are being explored, which are expected to significantly drive the Sickle Cell Disease treatment market during the forecast period.

How many companies are developing drugs for Sickle Cell Disease?
 
Key pharma players in Sickle Cell Disease market
The companies across the globe, such as GBT Therapeutics, Pfizer, Novartis, AstraZeneca, Ironwood Pharmaceuticals, Imara Therapeutics and many others that are involved in developing therapies for Sickle Cell Disease. The expected launch of emerging therapies, such as Voxelotor/ GBT-440 (GBT Therapeutics), Rivipansel (Pfizer), Crizanlizumab (Novartis), IMR-687 (Imara Therapeutics) and other compelling treatments are expected to increase the Sickle Cell Disease market size in upcoming years.



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