The global market is projected to expand due to the rising prevalence of urea cycle diseases. Urea cycle problems are anticipated to occur in 1 in 8500 births, according to statistics released by the Centres for Disease Control and Prevention (CDC). Governments from various countries and business organisations have been making a number of efforts in recent years to support the market's expansion. The market is being supported by growing initiatives such increased awareness campaigns, clinical trials and substantial research activities, as well as investments in R&D and easy regulatory clearances. For instance, Mead Johnson Nutrition Company (MJN) and Reckitt Benckiser Group plc (RB) announced their merger in 2017.
During the projected period of 2023–2027, it is anticipated that the global U.S. Urea Cycle Disorders Treatment Market would expand at a remarkable rate.
Leading Companies in the U.S. Urea Cycle Disorders Treatment Industry:
1. Bausch Health Companies Inc.: Established in 1959. Headquarters in Canada. The dermatological, gastrointestinal, and ophthalmology industries are the focus of the worldwide speciality pharmaceutical, consumer health, and medical device corporation Bausch Health Companies Inc. Salix, International, Solta Medical, Diversified Products, and Bausch + Lomb are the company's five revenue-reporting sectors. The segment that brings in the most money is Bausch + Lomb. Recent purchases include Unilens Vision (July 2015) and Paragon Holdings (Feb 2017).
2. Recordati Rare Diseases: Constructed in 1926. Italian headquarters are in Milan. A pharmaceutical manufacturer with a speciality in specialised drugs is Recordati SpA. The company develops and markets medications with a global focus on primary medical treatments and uncommon disorders. Strategic collaborations are a part of Recordati's long-term business strategy. Italy generates the great bulk of the company's revenue, followed by the US, France, and Germany. The two operating segments reported by Recordati are specialty and primary care and rare diseases. The company's specialised and primary care business, which also involves the creation of active substances, generates the majority of its profits. To increase the variety of treatments it offers for rare diseases, Recordati has agreed to purchase EUSA Pharma from a third party for an estimated $848.9 million.
3. Eurocept Pharmaceuticals Holding (Lucane Pharma SA): Established in 2001. Headquarters in the Netherlands at Ankeveen. A company that offers medical homecare and medications with the goal of streamlining the patient-physician therapeutic alliance. The business helps clients with their needs for pharmaceutical, medical-technical, and nursing care by offering services in the areas of distribution, production, registration, marketing, selling of medicinal products, hospital account management, and pharmacovigilance. In order for Eurocept B.V. to acquire 100% of Lucane Pharma, Dechert LLP provided assistance.
4. Acer Therapeutics: Developed in 2013. Headquarters in the United States. Pharmacies are produced by Acer Therapeutics Inc. The acquisition, development, and commercialization of treatments for people with severe rare and ultra-rare diseases who have a serious unmet medical need are its main areas of concentration. Acer Therapeutics Inc., a pharmaceutical company specialising in the acquisition, development, and commercialization of treatments for serious, rare, and life-threatening diseases with significant unmet medical needs, announced today that it has entered into a binding agreement to buy and sell 2,920,306 shares of the Company's common stock in a registered direct offering at a price of $0.916 per share (or the equivalent in common stock).
5. Ultragenyx Pharmaceutical: Established in 2010. Headquarters in the United States. An American biopharmaceutical business called Ultragenyx Pharmaceutical Inc. With a focus on severe, crippling genetic disorders, it finds, acquires, develops, and commercialises innovative therapies for the treatment of severely rare and ultra-rare diseases. Crysvita, Dojolvi, and Mepsevii are all included in the company's portfolio of medications. For the treatment of X-linked hypophosphatemia (XLH) in adult and paediatric patients 1 year of age and older, crysvita is recommended. Mepsevii is recommended for the treatment of mucopolysaccharidosis VII in both paediatric and adult patients.
6. Aeglea BioTherapeutics: Developed in 2013. United States headquarters. Aeglea BioTherapeutics Inc. is a biotechnology business in the clinical stages of creating human enzyme therapies for persons with uncommon metabolic illnesses. The business competes in the areas of biotechnology, pharmaceuticals, and other associated markets that deal with rare genetic illnesses. Spyre Therapeutics to be acquired by Aeglea BioTherapeutics.
7. Arcturus Therapeutics, Inc.: Established in 2013. United States headquarters. An RNA medicine firm with a present focus on liver and respiratory disorders, Arcturus Therapeutics Holdings Inc. looks for prospects in rare diseases. It works in the area of research and development for technology that focuses on nucleic acids and has medical applications. The company's product pipeline consists of LUNAR-OTC, LUNAR-CF, and LUNAR-COV19. Both Arcturus Therapeutics, Inc. and Alcobra Ltd. accept the merger.
*Definition: The treatment of urea cycle disorders (UCDs) typically requires a team of medical professionals. This may include specialists at a metabolic center. Specialists can include a metabolic geneticist, a metabolic dietitian, a genetics counselor, a nurse practitioner, and a social worker who have expertise in treating UCDs.
