Orphan Product and Supplier Directory

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...

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
...

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...

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.

Tisinon (Nitisinone) 5 mg, 10 mg, 20 mg Capsule

Product Nitisinone Menagen

Indicated for hereditary tyrosinemia type 1

Product Deutivacaftor

Deutivacaftor, also known as ivacaftor-d9, VX-561, or CTP-656, is a precision-deuterated analog of ivacaftor in which nine hydrogen atoms are replaced by deuterium. It is a CFTR potentiator that increases the probability of channel opening of CFTR proteins delivered to the cell surface. Deuteration slows d...

Product Deferoxamine mesylate

The API will be used for the development of generic version of Desferal injection

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...

Product Biosimilars, orphan drugs, Oncology, Vaccines, Blood products, Narcotic & Psychotropic Drugs

Biosimilars, orphan drugs, Oncology, Blood products, Narcotic & Psychotropic Drugs

Biosimilars, orphan drugs, Oncology, Vaccines, Blood products, Narcotic & Psychotropic Drugs

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.

Tisinon (Nitisinone) 4 mg/1 mL Oral Suspension

Product Vanzacaftor

Vanzacaftor (VX-121) is a CFTR corrector for class II CFTR mutations, the most common being F508del. It improves the processing and trafficking of defective CFTR proteins, allowing them to reach the cell surface and function properly. Vanzacaftor is administered as part of a triple combination with tezacaf...

Product Ivacaftor

Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. It binds directly to the CFTR protein at the cell surface and increases the open probability of the ion channel, thereby enhancing chloride transport across cell membranes. It is indicated for the treatment of cystic fib...

Product Tezacaftor

Tezacaftor is a CFTR corrector. It facilitates the cellular processing and trafficking of normal and select mutant forms of CFTR (including F508del-CFTR) to increase the amount of mature CFTR protein delivered to the cell surface, resulting in increased chloride transport. Tezacaftor is used in combination...

Product Elexacaftor

Elexacaftor is a CFTR corrector that binds to the F508del-CFTR protein and stabilizes its conformation, thereby facilitating its processing and trafficking to the cell surface. When combined with tezacaftor and ivacaftor, elexacaftor and tezacaftor bind to different sites on the CFTR protein and have an ad...

Product L-Glutamine oral powder

Developed in India, a generic version of Endari approved in US

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...

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....