Tag Archives: oprhan drug market

FDA grants orphan drug status to SER100 in PAH

Serodus announces orphan drug designation for its leading product, SER100 by US Food and Drug Administration (FDA) for the treatment of Pulmonary Arterial Hypertension (PAH). The company is planning for first clinical study in phase IIa or combined phase IIa/III study in patients with PAH.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

SB- Fix receives orphan drug designation for Hemophilia B

 FDA announces orphan drug designation status to SB-FIX , zinc finger nuclease (ZFN)-mediated genome editing product for the treatment of hemophilia  from Sangamo Biosciences. The company has aces in therapeutic genome editing.

SB-FIX is designed as a single treatment strategy intended to provide stable, continuous production of Factor IX clotting protein (FIX) for the lifetime of the patient. Sangamo’s ZFN-mediated in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic “safe-harbor site,” that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient’s liver to permanently produce circulating therapeutic levels of a corrective protein product.

Hemophilia B is a rare bleeding disorder caused by mutations in genes that encode factors which help the blood clot and stop bleeding when blood vessels are injured. The patients of Hemophilia B experience continuous bleeding after injuries which leads to joint diseases such as arthritis.

Rare diseases are also known as Orphan diseases. These diseases are generally categorized as chronic, degenerative, and life-threatening. The rare diseases normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders, which are extremely life-threatening. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. These two regions form a dominant portion of the global market for orphan drugs. Orphan drug status is intended to facilitate drug development for rare diseases and provides several benefits to drug developers, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com
Or Visit:  Global Orphan Drug Market Future Outlook 2020

 

FDA grants BioXcel’s BXCL101 orphan drug designation for Neurofibromatosis Type 2 (NF2)

BioXcel, a private pharmaceutical company in Connecticut, declares tht FDA has granted BXCL101, orphan drug designation status for the treatment of the patiets suffering from Neurofibromatosis Type 2, disease associated with neurologic and ophthalmologic abnormalities caused by benign tumors of the brain, spinal cord and peripheral nerves.

BXCL101 is a proprietary version of an approved drug, bortezomib, adapted for chronic use in NF2 patients with both a novel dosing regimen and delivery approach.  NF2 is the first drug discovered on BioXel’s R&D platform to be granted orphan drug designation.

Rare diseases are also known as Orphan diseases. These diseases are generally categorized as chronic, degenerative, and life-threatening. The rare diseases normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders, which are extremely life-threatening. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. These two regions form a dominant portion of the global market for orphan drugs.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020