Orphan
Orphan Companies (18)
Orphan News
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News CPHI Podcast Series: Investing in a vision for the future of life sciences
In this episode Lucy Chard is joined by Rajiv Khatau to discuss the importance of looking into new therapeutic areas and some of the more niche areas of pharmaceuticals, and investing in the future of the industry.
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News First treatment for Acid Sphingomyelinase Deficiency approved by US FDA
Enzyme replacement therapeutic Xenpozyme has received US FDA approval with an orphan drug designation to treat symptoms of Acid Sphingomyelinase Deficiency, a rare genetic disease.
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News Rare diseases care pathway: a major challenge to harmonize patient management
In France, rare diseases are numerous: between 7,000 and 8,000 diseases are currently accounted for, and concern more than 3 million French people, accounting for almost 4,5% of the total population. The organization of screening and care for these ill...
Orphan Products (12)
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Product CGMP Contract laboratory services
Our mission is to deliver outstanding pharmaceutical services at affordable prices, with an emphasis on speed, accuracy, and clear communication, ensuring every client receives dependable results and a seamless experience.
We invite attendees to visit Booth 915 to discuss how CRC Labs can...
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Product EZ-fill® Large Volume Cartridges
Designed for the delivery of high-volume, high-viscosity biologics, EZ-fill® Large Volume Cartridges support treatments for several conditions such as autoimmune, oncology, rare diseases and Parkinson’s.
With 10+ years of expertise in large-volume containers, we are a trusted partner for...
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Product KD Pharma lipids APIs and Intermediates
Lipid Portfolio
Omega-3 APIs
· Largest producer of omega-3 APIs worldwide
· Ultra-high purity: EPA up to 99%, DHA up to 95%, tailored ratios
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Product Sodium Oxybate
Sodium Oxybate is the sodium salt of gamma-hydroxybutyric acid (GHB), classified as an orphan drug for the treatment of narcolepsy. It is a central nervous system depressant with proven efficacy in reducing cataplexy and excessive daytime sleepiness. DAPIN holds a valid EU Active Substance Master File (ASM...
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Product Tisinon (Nitisinone) 5 mg, 10 mg, 20 mg Capsule
Nobel Ilac Sanayii Ve Ticaret A.S provides wide range of pharmaceutical products which includes Tisinone
Active ingredients: Nitisinone
Indication: Hereditary Tyrosinemia Type 1
Contact us for more information.
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Product Deferoxamine mesylate
The API will be used for the development of generic version of Desferal injection
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Product Octreotide Acetate Injection
Octreotide injection is used to treat severe diarrhea and other symptoms that occur with certain intestinal tumors (eg, vasoactive intestinal peptide tumors or VIPomas) or metastatic carcinoid tumors (tumors that has already spread in the body).
It is also s used to decrease the amount...
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Product Tisinon (Nitisinone) 4 mg/1 mL Oral Suspension
Nobel Ilac Sanayii Ve Ticaret A.S provides wide range of pharmaceutical products which includes Tisinone
Active ingredients: Nitisinone Indication: Hereditary Tyrosinemia Type 1
Contact us for more information.
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Product Enabling access to orphan & rare therapies in Bulgaria
BB Pharm is a Bulgarian pharmaceutical wholesaler specialized in rare and orphan therapies. We connect international manufacturers with patients in Bulgaria through a fully independent and compliant access model. Our services include regulatory coordination, sourcing, distribution, and validated logistics....
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Product Biosimilars, orphan drugs, Oncology, Vaccines, Blood products, Narcotic & Psychotropic Drugs
Biosimilars, orphan drugs, Oncology, Blood products, Narcotic & Psychotropic Drugs
Upcoming Events
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CPHI & PMEC China 2026
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25 Aug 2026 - 27 Aug 2026


