SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other pulmonary diseases, today announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted orphan drug designation (ODD) to SPL84-23-1, an antisense oligonucleotide (ASO) targeting the 3849 mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene associated with a severe form of CF lung disease.
Cystic fibrosis – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030
According to a literature survey, Each year in the United States, ~1,000 to 1,250 new cases are being reported. Approximately 30,000 to 37,000 cases of Cystic Fibrosis are being present in the USA. According to Thelansis Research, around 97% of cases of Cystic Fibrosis are being reported in the USA whereas in the EU countries the diagnosis rate is less than 75%. More than 95% of male patients are infertile and ~50% of female patients are infertile.
Comprehensive insight on patient segmentation based on age, sex, Types (Typical & Atypical), Genotype (CFTR, ENaC), Severity (mild, moderate & severe), Clinical manifestations (Pulmonary Manifestations, Head and Neck Manifestations, Pancreatic Manifestations, Hepatobiliary Manifestations, Gastrointestinal Manifestations, Bone/ Tissue disease, and Reproductive System Manifestations, and Others), Treatment types (Lines of Therapy) has been provided in-to the epidemiology section of the Cystic Fibrosis (CF) and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China.
In terms of pharmacologic therapies, there are few drug candidates are in different Phase (Phase I, II & III) stages of development. Key companies are, Horizon Pharma, Vertex Pharmaceuticals Incorporated, PTC Therapeutics, Insmed Incorporated, Forest Laboratories, Anthera Pharmaceuticals, Chiesi Farmaceutici S.p.A., AlgiPharma AS, Zambon SpA, Abbott, Anthera Pharmaceuticals, Novartis Pharmaceuticals, Novartis Pharmaceuticals, Galapagos NV, and Beyond Air are the key companies focus to the Cystic Fibrosis market.
Thelansis is specialized in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area which includes both rare / ultra-rare and mainstream indication. Over the period of time, we have built a strong repository of 6,000+ Bio-pharma reports which essentially covers Epidemiology study and Market forecasting based on the KOL opinions. Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the major focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.
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