Tag Archives: orphan drug overview

Novel Hepatocellular Carcinoma Tx receives orphan drug status by FDA

U.S Food and Drug Administration (FDA) have granted orphan drug designation to Yisheng Biopharma’s leading candidate, hepatocellular carcinoma treatment, YSON-001. The drug is complex in nature with multi-component structure of broad immune-modulating properties such as promoting Th1-biased immunity, inducing the activation and proliferation of dendritic cell, B and natural killer cells, promoting macrophage M1 polarization and down regulating regulatory T cells.  The treatment is clinical biological product or immune-modulating technology derived from Yisheng Biopharmaceutical Company.

Orphan Drug designation is granted to those medications or therapy that are rare, more specifically, affect less than 200,000 patients in the country. These rare diseases are incurable and severe in nature and may cause death. To fulfill the need of treat options for individuals, FDA grants orphan drug designation to drugs which are investigated to be helpful in the treatment of such rare diseases. Orphan drug status is a package of several financial benefits for the drugs and its manufacturers such as certain tax credits and exemption from paying prescription drug user fees normally required from companies submitting products for approval.

 

 

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

 

 

FDA grants orphan drug status to ARMO’s AM0010

ARMO Biosciences Inc., a clinical stage biotechnology company declared that it’s leading immune-oncology drug, AM0010 (PEGylated Interleukin-10), has been granted orphan drug designation by U.S Food and Drug Administration for the treatment of pancreatic cancer. The company has been granted with fast track designation for AM0010 in combination with FOLFOX as a second line therapy in patients with advanced metastatic pancreatic cancer.

Over 340 patients with advanced solid malignancies have been investigated in the ongoing phase 1 trial, either as a single agent or in combination with standard-of-care chemotherapy drugs or anti-PD-1 antibodies. The company is planning to initiate a pivotal Phase 3 trial by the late 2016, using AM0010 in combination with FOLFOX as a second-line therapy in patients with advanced metastatic pancreatic cancer.

Orphan Drug designation is granted to those medications or therapy that are rare, more specifically, affect less than 200,000 patients in the country. These rare diseases are incurable and severe in nature and may cause death. To fulfill the need of treat options for individuals, FDA grants orphan drug designation to drugs which are investigated to be helpful in the treatment of such rare diseases. Orphan drug status is a package of several financial benefits for the drugs and its manufacturers such as certain tax credits and exemption from paying prescription drug user fees normally required from companies submitting products for approval.

 

 

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

EMA announces orphan drug designation for ABO-102 gene therapy

Abeona Therapeutics Inc., a biopharmaceutical company dedicated to develop gene therapies for life threatening rare diseases declared that leading therapy ABO-102 has been designated as orphan drug b European Medicines Agency (EMA) for the treatment of Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease causing neurocognitive decline, speech of loss, premature death in children and loss of mobility.

Abeona’s MPS IIIA program has been previously granted the designation of orphan drug by Food and Drug Association (FDA) in the US and received the Rare Pediatric Disease Designation as a pre-requisite part of the Priority Review Voucher (PRV) process.

The FDA and EMA’s designation of orphan status is granted to encourage new developments and therapies for rare diseases and disorders. Orphan Status can be applied to products proven safe and effective in treating conditions that affect a relatively small number of patients, and the designation may entitle up to seven years of US marketing exclusivity upon regulatory approval. Companies whose drugs have an orphan designation also receive certain tax credits and are exempted from paying prescription drug user fees normally required of companies submitting products for approval.

 

 

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