The Orphan Drug Act passed in 1983 and proved to be an extremely exciting time for Americans with rare diseases and their families. The Orphan Drug Designation Program is the most crucial part of the legislation as it provides vital financial incentives to encourage organizations and companies to develop drugs and biologics for rare diseases. The legislation provides tax cuts to help cover the cost of clinical trials and gives eligibility for 7 years of market exclusivity. The number of requests for orphan drug designation (ODD) continues to increase and has grown significantly. In 2014, there was a 30% increase other the prior year’s record number. The pace does not seem to be slowing down at all making it crucial that investors and medical professionals alike understand how to distinguish the most valuable ODD’s from others.


A rare disease is defined as “a condition that affects fewer than 200,000 people living in the U.S.” The U.S. has a population of almost 320 million people, meaning there are roughly 30 million Americans who suffer from a rare disease. A few of the latest ODD’s include Cystradrops, Dinutuximab beta Aperion, Ledaga, and PRP.

Cystradrops comes from Orphan Europe S.A.R.L. and is an eye medicine used in patients from two years of age and up who have cystinosis, an inherited disease where cysteine builds up forming damaging crystals. Dinutuximab beta Aperion developed by Apeiron Biologics AG is a cancer medicine used to treat neuroblastoma, a cancer of nerve cells. Ledaga developed by Acetelion Registration Ltd. treats adults with skin cancer (mycosis fungoides-type cutaneous T-cell lymphoma). Propanc Biopharma Inc.’s ODD is for their lead product, PRP, is a solution for once daily intravenous administration of a combination of two pancreatic proenzymes trypsinogen and chymotrypsinogen, for the treatment of pancreatic cancer.

The four aforementioned drugs treat rare disease and provide hope for suffering patients. Investors and medical professionals should carefully assess the severity of each disease an ODD treats when investing or promoting products. Three of the four ODD’s in this article treat different types of cancer but only PRP treats pancreatic cancer.  Pancreatic cancer is one of the deadliest, if not the deadliest, cancer. This diagnosis gives patients a median survival rate of 6 months and less than 5% of patients live up to 5 more years. While a whole host of companies receive ODD’s, PRP is the most valuable as it treats pancreatic cancer.


On June 1st, Moleculin Biotech, Inc. (MBRX) announced the launch of its investor website at Investors may visit this website for information about the Company, its SEC filings, press releases and other information of interest to current and potential investors.

Esperion Therapeutics, Inc. (ESPR) announced earlier this week the FDA confirmed the regulatory pathway to approval for the once-daily, oral combination pill of bempedoic acid 180 mg and ezetimibe 10 mg.

In May, Juno Therapeutics (JUNO) announced it has appointed Jay Flatley to its Board of Directors. Mr. Flatley is Executive Chairman of Illumina, Inc.

Arena Pharmaceuticals, Inc. (ARNA) announced today their completion of a Phase 1 comparative bioavailability study evaluating the pharmacokinetic (PK) profile of extended release (XR) ralinepag compared to the immediate release (IR) formulation in healthy adults.

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