Prader-Willi syndrome (PWS) is a genetic disorder due to the deletion of a part of the father's chromosome 15. Prader-Willi syndrome can also be due to the presence of two copies of chromosome 15 inherited by the mother only, leading to their suppression while the gene formation. PWS therapeutics has a wide scope in the global market, as there is no known cure available for such a complex genetic disorder.
Market Dynamics
The presence of a strong pipeline is expected to drive the market over the forecast period. For instance,
Company Drug Study title Phase Anticipated launch
Soleno Therapeutics, Inc. DCCR(diazoxide choline controlled-release tablets) A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome Phase 3 December 2019
Millendo Therapeutics, Inc. Livoletide Prader-Willi Syndrome Phase 2 March 2021
Levo Therapeutics, Inc. carbetocin Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome (CARE-PWS) Phase 3 December 2020
Montefiore Medical Center in collaboration with Foundation for Prader-Willi Research cannabidivarin (CBDV) CBDV versus Placebo in Children With Prader-Willi Syndrome (PWS) Phase 2 October 2020
INSYS Therapeutics Inc Cannabidiol Oral Solution A study to evaluate the safety of pharmaceutical-grade synthetic cannabidiol oral solution in patients with prader-willi syndrome Phase 2 May 2020
However, limited efficacy of the current therapies, lack of awareness of the prader-willi syndrome, and the presence of complications associated with the diseases are expected to restrain the market growth.
The clinical complications frequently found in PWS include excessive daytime sleepiness, scoliosis, osteopenia/osteoporosis, decreased gastrointestinal motility, sleep disturbances, and reduced pain sensitivity.
Based on the product type, the global prader-willi syndrome market is segmented into Beloranib, Betahistine Hydrochloride, Diazoxide Choline CR, FE-992097, recombinant growth hormones, and others.
The recombinant growth hormones segment is expected to grow at a high rate over the forecast period. At present, the pharmacological management of prader-willi syndrome (PWS) focuses mainly on the use of human growth hormone (HGH) supplementation, which is approved in the US. Growth hormone(GH) supplementation constitutes the primary component of the entire therapeutic market composition due to the nearly ubiquitous deficiency of growth hormone (GH) in PWS patients. Some of the majorly approved GH therapies include Pfizer's Genotropin (somatropin) along with other major brands such as Norditropin (Novo Nordisk) and a few others in the US. Another approved drug is Omnitrope (somatropin [rDNA origin] injection) from Sandoz. Also, the presence of ongoing clinical trials on growth hormone is expected to boost the market growth. For instance,
Company Drug Indication Phase Anticipated launch
GeneScience Pharmaceuticals Co., Ltd. Recombinant Human Growth Hormone (rhGH) Injection Prader-Willi syndrome Phase 3 January 2020
The beloranib segment is also expected to drive the market growth over the forecast period, owing to the presence of research and development activities. For instance, according to the lead investigator Dr. Merlin G. Butler, at the annual meeting of the Endocrinology Society, reported that, in 2016, Prader-Willi syndrome (PWS) patients lost more than 4% of the body weight over a 26-week period with the investigative anti-obesity drug beloranib, compared with a similar gain in patients taking placebo, in phase 3 best PWS trial. Further, Hunger-related behavior was also decreased, along with total and LDL cholesterol levels and other cardiometabolic risk factors. Thus, the research and development activities on beloranib is driving the market.
The betahistine segment is expected to grow at a high rate over the forecast period, owing to the presence of the key players manufacturing betahistine. For instance, on December 6, 2018, Auris Medical Holding AG, a clinical-stage company dedicated to developing therapeutics that address critical unmet needs in neurotology and the mental health supportive care, has announced a strategic expansion for the intranasal betahistine development program. In 2 related transactions, the company has acquired an orphan drug designation for betahistine in the treatment of obesity-associated with Prader-Willi syndrome (PWS) and signed a binding letter of intent to in-license exclusive rights to 2 US Patents relating to the use of betahistine for the treatment of depression and attention-deficit / hyperactivity disorder (ADHD), respectively.
Geographically, the global prader-willi syndrome market is segmented into five major regions: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa.
North America is dominating the global prader-willi syndrome market in 2018 and is estimated to hold significant market size over the forecast period (2019-2026) owing to the increasing prevalence of prader-willi syndrome in the region, a growing number of ongoing research and development activities on the prader-willi syndrome.
The presence of organizations which are creating awareness about PWS in the region is also expected to drive the market growth. For instance, the Prader-Willi Syndrome Association (USA) has been providing life-saving research, crisis, and family support, medical and new parent support since 1975.
The increasing number of ongoing clinical trials on the prader-willi syndrome in the region is expected to drive the market growth over the forecast period. For instance,
Montefiore Medical Center Intranasal Oxytocin (IN-OXT) Prader-Willi Syndrome Phase 2 August 2020
Indiana University Behavioral: Listening Project Protocol Prader-Willi Syndrome Not applicable May 2020
Competitive Landscape
The key players operating in the global prader-willi syndrome market are Novartis, Soleno Therapeutics, Saniona, Pfizer, Millendo Therapeutics, Inc., Levo Therapeutics, Inc., Rhythm Pharmaceuticals, Essentialis, Inc., and Novo Nordisk.
The key players are adopting various strategies such as product launches, mergers & acquisitions, partnerships, and collaborations, which are contributing to the growth of the prader-willi syndrome market globally. For instance,
In September 2019, Saniona reported positive Phase 2a clinical results in adolescent patients with prader-willi syndrome.
On December 27, 2016, Capnia, Inc. and privately-held Essentialis, Inc., announced their entry into a definitive agreement. The company will be able to advance the diazoxide choline controlled-release (DCCR) tablet, a once a daily oral tablet for the treatment of patients with prader-willi syndrome.
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