Tag Archives: orphan drug market regulation

Amryt receives orphan drug status for Episalvan

Food and Drug administration (FDA) has declared orphan drug designation for Amryt Pharma’s leading treatment therapy Episalvan for the treatment of epidermolysis bullosa (EB). It is a rare and distressing genetic disorder affecting young children to have a highly fragile skin. Currently, the pharmaceutical company is planning to carry out Phase-3 clinical trial to assess its potential to accelerate the healing process in emidermolysis bullosa.

The firm claims that the product is already available in the market to treat gash or ulcers also referred as partial thickness wound in adults. Episalvan is a skin treatment therapy for kids and adults. The results from other clinical trials have been encouraging. The skin treatment therapy has been awarded with the orphan drug status in United States by FDA and Europe by European Commission.

Orphan drug designation is granted by Food and Drug administration and European Commission to the drugs and treatments or therapies that claims to have the cures of rare diseases. These rare diseases are defined as the diseases affecting less than 20,000 people in a country. These diseases have do not have common treatment methods or other medicinal cures and are incurable and severe in nature which may cause death. FDA and EU Commission address the need of such patients suffering from rare diseases by granting orphan drug status to these medications and treatments. Additionally, these authorities provide certain incentives and relaxation in the fees to the companies producing these drugs.

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

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

 

 

AbbVie receives orphan status for ABBV-006 in U.S

The U.S Food and Drug Administration have granted orphan drug designation to ABBV-006, developed by AbbVie pharmaceuticals for the treatment of Crohn’s Disease in pediatric patients. The drug is currently in its investigational phase, IL-23 inhibitor, rizankumab, (ABBV-066; formerly BI 655066).

In March, 206, AbbVie and Boehringer Ingelheim collaborated for an agreement stating that the former is responsible for the commercialization and development of Rizankumab globally. AbbVie acquired all rights to rizankumab under this collaboration agreement. Currently, rizxankumab is also under investigation for its use in the treatment of immunological disorders such as psoriasis, psoriatic arthritis and asthma.

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 PRX-OTC by PhaseRx

PhaseRx, a biopharmaceutical company announced that its leading product, PRX-OTC has been granted orphan drug designation for the treatment of ornithine transcarbamylase deficiency by U.S Food and Drug Administration (FDA).

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

European Union grants orphan drug status to Crenolanib

Arog Pharmaceuticals declared that its leading drug candidate has received orphan drug designation for the treatment of patients with acute myeloid leukemia and soft tissue sarcoma.

 The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. The orphan medicinal product designation provides the company and its drug with certain benefits and incentives in the EU, including a period of market exclusivity of 10 years after the market approval, free consultation and scientific advice on contact with European Medicinal Agency.

 

 

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