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GX-H9 receives orphan designation for growth hormone deficiency

The Food and Drug Association has granted orphan drug designation to Generix’s leading product, GX-H9, for the treatment of patients with growth hormone deficiency.

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

Or Visit:  Global Orphan Drug Market Future Outlook 2020

 

FDA announces orphan drug designation for Veliparib as NSCLC treatment

US Food and drug Association (FDA) has declared Veliparib, an oral poly (adenosine diphosphate [ADP]-ribose) polymerase (PARP) inhibitor, developed by Abbvie (ABBV). Currently, Veliparib is undergoing investigation in combination with chemotherapies, such as carboplatin and paclitaxel, or radiation for the treatment of advanced squamous non-small cell lung cancer (NSCLC).

PARP is the naturally occurring enzyme in the body that repairs damage to DNA in the cells. The process is useful to maintain the integrity of healthy cells, and can help in repairing DNA cancer cells, causing them to survive. Veliparib is currently under investigation that its combination with DNA damaging therapies, such as chemotherapy or radiation, may lessen the repair of DNA damage in cancer cells, eventually causing some cells to die.

NSCLC is the common type of lung cancer in the US accounting for 80 to 85% of diagnosed cancer cases. Survival from lung cancer depends on the stage at which cancer is diagnosed.

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