Approval and Launch of New Drugs and R&D of New Therapies to Augment Growth of Cystinosis Treatment Market

 


Cystinosis is an inflammatory condition characterized by excess accumulation of the harmful amino acid casein (a building block of amino acids) in tissues and organs. Excessive cystinosis often forms crystals that can settle into tissues and organs and cause serious problems in many bodily systems and organs. It is also accompanied by low red blood cell count, increased blood pressure, and headaches. In most patients, cystinosis clears after several years. The condition is usually inherited and develops slowly over time.

Market Dynamics

Approval and launch of new drugs is expected to propel the growth of the cystinosis treatment market. For instance, in March 2020 AVROBIO, Inc. received the U.S. Food and Drug Administration’s orphan drug designation for its investigational gene therapy, AVR-RD-04, for the treatment of cystinosis.

R&D of new therapies is expected to offer lucrative growth opportunities for players in the cystinosis treatment market. For instance, in January 2021, Eloxx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, announced that a scientific manuscript titled: “Phase 1 Renal Impairment Trial Results Enable Targeted Individualized Dosing of ELX-02 in Nephropathic Cystinosis Patients” has been published in the Journal of Clinical Pharmacology. Moreover, approval for the novel therapy treatments for cystinosis is also expected to aid in the growth of the market. For instance, in January 2019, The Cystinosis Research Foundation, provider of grants for cystinosis research, announced that the Federal Drug Administration (FDA) granted approval for the first human clinical trial for stem cell and gene therapy treatment for cystinosis.

Among regions, North America is expected to witness significant growth in the cystinosis treatment market, owing to approval and launch of new products. For instance, in April 2020, Horizon Therapeutics plc announced that PROCYSBI (cysteamine bitartrate) delayed-release oral granules in packets are now available in the The United States for adults and children one year of age and older living with nephropathic cystinosis.

Major players operating in the cystinosis treatment market are focused on R&D to expand their product portfolio. For instance, in August 2020, Recordati Rare Diseases received the U.S. FDA approval for cysteamine ophthalmic solution 0.37% for the treatment of patients living with cystinosis.

Competitive Analysis

Major players operating in the cystinosis treatment market include, Avrobio, Inc., Gilead, Mylan N.V., Novartis AG, Eloxx Pharmaceuticals, Inc., Horizon Therapeutics plc, Recordati Rare Diseases, and Orizon Pharma Plc.

Major players operating in the cystinosis treatment market are focused on R&D to expand their product portfolio. For instance, in July 2020, AVROBIO Inc. announced that a patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.

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