Top Companies Receiving Orphan Drug Designation in 2017

Friday, July 28, 2017 Drug News
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LAS VEGAS, July 28, 2017 /PRNewswire/ --

Under the Orphan Drug

Act (ODA), drugs, vaccines, and diagnostic agents qualify for orphan status if they are intended to treat a disease affecting less than 200,000 American citizens. Orphan drug sponsors qualify for seven-year FDA-administered market Orphan Drug Exclusivity
(ODE), tax credits of up to 50% of R&D costs, R&D grants, waived FDA fees, protocol assistance, and may get clinical trial tax incentives. This designation helps crucial drug treatments move to the medical market faster, increasing the chance to save lives, whilst also providing exclusivity in the marketplace for their drug which maximizes their returns.

Here are a few companies that have recently been approved for Orphan Drug Designation: Propanc Biopharma Inc. (OTC: PPCB), Abeona Therapeutics Inc. (NASDAQ: ABEO), Mallinckrodt plc (NYSE: MNK), Conatus Pharmaceuticals (NASDAQ: CNAT), Epizyme Inc. (NASDAQ: EPZM) and Zogenixm Inc.

About the latest Orphan Drug Sponsors: 

Propanc Biopharma Inc. (OTCQB: PPCB), a clinical stage biopharmaceutical company focusing on development of new and proprietary treatments for cancer patients suffering from solid tumors such as pancreatic, ovarian and colorectal cancers, a few weeks ago announced the Company received Orphan Drug Designation (ODD) from the FDA for the use of its lead product, PRP, a solution for once daily intravenous administration of a combination of two pancreatic proenzymes trypsinogen and chymotrypsinogen, for the treatment of pancreatic cancer. The approved indication is one of the most lethal malignancies with a median survival of 6 months and a 5-year survival rate of less than 5%. The lethal nature of this disease stems from its propensity to rapidly disseminate to the lymphatic system and distant organs, and is a major unmet medical issue.

Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, in January announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation for Abeona's ABO-201 program (AAV-CLN3), the AAV-based single intravenous gene therapy program for juvenile Batten disease, a fatal lysosomal storage disease of the nervous system caused by autosomal-recessive mutations in the CLN3 gene.

Mallinckrodt plc (NYSE: MNK), a leading specialty pharmaceutical company, recently announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MNK-1411, a long-acting formulation containing cosyntropin acetate under investigation for the treatment of Duchenne muscular dystrophy (DMD)

Conatus Pharmaceuticals Inc. (NASDAQ: CNAT) announced in June that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Conatus' drug candidate IDN-7314 for the treatment of primary sclerosing cholangitis (PSC), a disease affecting bile ducts in the liver which can lead to cirrhosis and liver failure. The FDA's Orphan Drug Designation program is intended to encourage the development of drugs and biologics that may provide benefit to patients suffering from rare diseases or conditions.

Epizyme, Inc. (NASDAQ: EPZM), a clinical stage biopharmaceutical company creating novel epigenetic therapies, announced in June that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to tazemetostat, the company's first-in-class EZH2 inhibitor, for the treatment of patients with soft tissue sarcoma (STS).

Zogenix, Inc., a pharmaceutical company developing therapies for the treatment of orphan and central nervous system (CNS) disorders, announced in June the U.S. Food & Drug Administration (FDA) has granted its investigational drug, ZX008 (low-dose fenfluramine), orphan drug designation for the treatment of Lennox Gastaut Syndrome (LGS), a refractory, debilitating childhood-onset epilepsy. The Orphan Drug Designation by the FDA follows the European Union's granting of Orphan Drug Designation for ZX008 in the treatment of LGS earlier this year.

These companies all have a significant advantage over their competitors receiving tax credits of up to 50% of R&D costs, R&D grants, waived FDA fees, protocol assistance, and may get clinical trial tax incentives.

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