Pharma Industry Poised for Fast Start in 2016 as U.S. Food & Drug Administration Receives Requests for Newest Orphan Drug Designations & Rare Disease Treatments

Dec 29, 2015, 08:30 ET from FN Media Group, LLC

CORAL SPRINGS, Florida, December 29, 2015 /PRNewswire/ --

Biotechnology companies continue to forge ahead with advancing clinical trials and development of next-generation treatments for rare diseases through new orphan drug designations and cutting edge drug delivery products. Today's companies in the spotlight with achievements and recent developments are Amarantus BioScience Holdings, Inc. (OTCQX: AMBS), pSivida Corp. (NASDAQ: PSDV), Adamas Pharmaceuticals, Inc. (NASDAQ: ADMS), Auris Medical Holding AG (NASDAQ: EARS) and Cerus Corporation (NASDAQ: CERS).

Amarantus BioScience Holdings, Inc. (OTC: AMBS), a biotechnology company focused on developing products for Regenerative Medicine, Neurology and Orphan Diseases, announced that it has requested Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) to treat GCMN with Engineered Skin Substitute (ESS). It is estimated that the incidence of GCMN, a rare dermatological condition present at birth, is between 8 and 80 births annually in the United States.

Read the full Amarantus (AMBS) Press Release at

The FDA defines a "rare pediatric disease" as a disease that affects fewer than 200,000 individuals in the U.S. primarily aged from birth to 18 years. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain expedited FDA review for any subsequent marketing application. Vouchers  may be sold or transferred by the recipient; in the last 6 months, 2 priority review vouchers have been sold for a combined $595M in cash.  The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.

In other Biotech developments to watch this week:  pSivida Corp. (NASDAQ: PSDV), a leader in the development of sustained release drug delivery products for treating eye diseases, today announced that it plans to file for EU marketing approval of Medidur™ for chronic non-infectious uveitis of the posterior segment of the eye (posterior uveitis) based on data from a single pivotal trial as a result of the high statistical significance achieved in its first Phase 3 clinical trial. The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) advised pSivida that, consistent with the published Points to Consider (PtC) of the European Agency for Evaluation of Medicinal Products, an application for a product treating a condition like posterior uveitis could be based on statistically compelling and clinically relevant results from just one pivotal trial. The MHRA recently provided this specific advice in formal minutes of a meeting with pSivida held on October 29, 2015 (prior to the Company's receipt of topline results for its first Phase 3 clinical trial). With those results now available, pSivida plans to confirm with the MHRA the plan to file for EU marketing approval of Medidur based on one trial.

Adamas Pharmaceuticals, Inc. (NASDAQ: ADMS) last week announced that its Phase 3 EASE LID clinical trial evaluating the company's investigational compound ADS-5102 (amantadine HCl) extended-release capsules for the treatment of levodopa-induced dyskinesia (LID) associated with Parkinson's disease met its primary endpoint.  Results from this randomized, placebo-controlled study showed a statistically significant reduction (p = 0.0009) in LID at 12 weeks for patients who received ADS-5102 versus placebo as assessed by the Unified Dyskinesia Rating Scale (UDysRS).  This represents a 23 percent reduction in LID for ADS-5102-treated patients compared to placebo.  The reduction in LID was maintained at 24 weeks (p = 0.0008), a key secondary analysis.

Auris Medical Holding AG (NASDAQ: EARS), a clinical-stage company dedicated to developing therapeutics that address important unmet medical needs in otolaryngology, in November announced enrolment of the first patient into the pivotal HEALOS clinical trial with AM-111. HEALOS will evaluate the efficacy, safety and tolerability of single dose intratympanic injections of AM-111 in the treatment of idiopathic sudden sensorineural hearing loss (ISSNHL; a.k.a. "sudden deafness"). The trial will enroll 255 patients suffering from acute severe to profound hearing loss within 72 hours from ISSNHL

Cerus Corporation (NASDAQ: CERS) announced last week that Rhode Island Blood Center (RIBC) has signed a purchase agreement for the INTERCEPT Blood System for platelets and plasma. Rhode Island Blood Center is the primary supplier of blood products to hospitals in Rhode Island, and also services hospitals in nearby Massachusetts and Connecticut. RIBC distributes approximately 12,500 platelet and 42,000 plasma units annually. "Continuing to layer on screening tests to detect emerging pathogens that pose a risk to patients puts an economic strain on blood centers. We have experienced this first hand with babesiosis, a serious tickborne disease common in Rhode Island," said Lawrence Smith, President and CEO of the Rhode Island Blood Center. "The opportunity to proactively decrease the risk of transfusion-transmitted diseases made implementing INTERCEPT a priority to continue to fulfill our mission of providing a safe, plentiful and cost-effective blood supply."

In other recent NASDAQ market news of importance:  Fontem Ventures, owner of market leading e-cigarette brand blu, and Vapor Corp. (NASDAQ: VPCO), a U.S. based distributor and retailer of vaporizers and e-liquids, in December announced they have reached a settlement agreement that resolves ongoing litigation in the USA. The settlement ends another of eight patent infringement cases originally brought by Fontem Ventures and Fontem Holdings 1 in March 2014 in the United States District Court for the Central District of California in relation to e-vapour technology.  Under the terms of the settlement, Fontem Ventures has granted Vapor Corp. a non-exclusive royalty-bearing global licence under the patents asserted in the litigation and certain other e-vapour technology related patents. The remaining settlement terms are confidential. is leading provider of third party publishing & news dissemination services.  If you would like more information regarding our news coverage solutions, please visit for more details.  Get an edge on the market with our Premium News Alerts that are FREE for a limited time at  Follow us on Facebook: and Twitter: 

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