x

RSS Newsfeeds

See all RSS Newsfeeds

Global Regions

United States ( XML Feed )

Jul 31, 2017 6:20 AM ET

Top Companies Receiving Orphan Drug Designation in 2017

Disclosure NewswireTM

iCrowdNewswire - Jul 31, 2017

LAS VEGAS —

 

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.

Microcap Speculators
Email: info@microcapspecualtors.com   
Phone: +1-702-720-6310
Website: http://microcapspeculators.com/

DISCLAIMER: Microcap Speculators (MS) is the source of the content set forth above. References to any issuer other than the profiled issuer are intended solely to identify industry participants and do not constitute an endorsement of any issuer and do not constitute a comparison to the profiled issuer. FN Media Group (FNM) is a third-party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated with MS or any company mentioned herein. The commentary, views and opinions expressed in this release by MS are solely those of MS and are not shared by and do not reflect in any manner the views or opinions of FNM. Readers of this Article and content agree that they cannot and will not seek to hold liable MS and FNM for any investment decisions by their readers or subscribers. MS and FNM and their respective affiliated companies are a news dissemination and financial marketing solutions provider and are NOT registered broker-dealers/analysts/investment advisers, hold no investment licenses and may NOT sell, offer to sell or offer to buy any security.

MS Disclosure: Except for the historical information presented herein, matters discussed in this article contain forward-looking statements that are subject to certain risks and uncertainties that could cause actual results to differ materially from any future results, performance or achievements expressed or implied by such statements. ACR Communication, LLC. which owns Microcapspeculators.com, is not registered with any financial or securities regulatory authority, and does not provide nor claims to provide investment advice or recommendations to readers of this release. ACR Communication, LLC. which owns, Microcapspeculators.com may from time to time have a position in the securities mentioned herein and may increase or decrease such positions without notice. For making specific investment decisions, readers should seek their own advice. ACR Communication LLC. which owns Microcapspeculators.com may be compensated for its services in the form of cash-based compensation or in equity in the companies it writes about, or a combination of the two. For Full Legal Disclaimer Please use this link: http://microcapspeculators.com/disclaimer/  

This release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. “Forward-looking statements” describe future expectations, plans, results, or strategies and are generally preceded by words such as “may”, “future”, “plan” or “planned”, “will” or “should”, “expected,” “anticipates”, “draft”, “eventually” or “projected”. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company’s annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and MS and FNM undertake no obligation to update such statements.

Media Contact:
FN Media Group, LLC
editor@financialnewsmedia.com
+1-954-345-0611

SOURCE Microcapspeculators.com

Contact Information:

FN Media Group, LLC
editor@financialnewsmedia.com
+1-954-345-0611

View Related News >