Written by Ι Stock Market Media Group Staff — October 14, 2014
New York, NY – Nuvilex, Inc. (OTCQB: NVLX) has officially introduced its advanced pancreatic cancer treatment to the U.S. Food and Drug Administration (FDA) in an application asking the agency to grant orphan drug status to its Cell-in-a-Box/ifosfamide combination for the rare disease.
The FDA’s orphan drug program designates 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. In the United States and Europe, where Nuvilex also applied for the orphan drug designation, it’s easier to gain marketing approval with an orphan drug or biologic. If Nuvilex is granted orphan drug status in the U.S. and Europe, the company would enjoy 7 and 10 years, respectively, of marketing its pancreatic cancer treatment without any competition.
This marketing exclusivity is one benefit that makes smaller biotechnology firms attractive to Big Pharma, and given the data that two earlier Phase I/II clinical trials using Nuvilex’s treatment produced, orphan drug status could be the final piece of the puzzle that makes the Maryland firm an acquisition target for Big Pharma.
In a Thomson Reuters report on orphan drugs, analysis shows that orphan drugs/biologics address a high unmet medical need, so these drugs/biologics benefit from a pricing structure that makes them commercially attractive to larger pharmaceutical companies. Thomson Reuters points to the orphan oncology drug Rituxan (Rituximab), which is the world’s second most profitable drug just behind mainstream blockbuster Lipitor, and it is expected to garner more than $150 billion in revenue over its lifetime, the majority of which is for orphan indications.
In early 2015, Nuvilex expects to use its treatment of Cell-in-a-Box combined with low doses of the anticancer prodrug ifosfamide in a late phase clinical trial to go head-to-head with Celgene’s (NASDAQ: CELG) treatment of Abraxane® plus gemcitabine, the current gold standard for advanced pancreatic cancer. The Cell-in-a-Box/ifosfamide treatment produced better survival times than both Celgene’s treatment and Eli Lilly’s (NYSE: LLY) single agent treatment of Gemzar® in those two earlier trials.
The company is also conducting pre-clinical studies to treat symptoms associated with a list of abdominal cancers, so Nuvilex’s treatment could end up having multiple orphan indications as well.
According to a Casey Research study of drug sales that are at risk due to patent expirations, in 2012, expiring patents totaled $35.1 billion in branded drug sales losses, and another $33.5 billion in losses is expected in 2015. These patent expirations along with the FDA’s long approval process for developing new mass market drugs are driving some larger biopharmaceutical firms to turn to rare diseases and the benefits that come with developing those drugs.
Pfizer (NYSE: PFE) started its Rare Disease Research Unit for the purpose of developing strategic partnerships to create novel treatments across the spectrum of rare diseases. Irish drug maker, Shire (NASDAQ: SHPG), purchased US-based ViroPharma for $4.2 billion last year after the smaller biotech received orphan drug status for its drug Cinryze. In a blockbuster deal, Big Pharma’s Sanofi-Aventis (NYSE: SNY) paid $20 billion to buy orphan drug maker Genzyme.
So, Big Pharma has come to realize that the impact of the smaller patient population for rare diseases is offset by the higher price demanded by orphan drugs. Thomson Reuter’s analysis of the market shows that the compound annual growth rate (CAGR) of the orphan drug market between 2001 and 2010 was an impressive 25.8 percent, compared to only 20.1 percent for a matched control group of non-orphan drugs. This data, combined with the increasing number of orphan drug approvals, suggests that the CAGR of launched orphan drugs will outshine that of the non-orphan control drugs over the next 30 years.
In September, Nuvilex applied for the orphan drug designation with the European Medicines Agency (EMA) for its advanced pancreatic cancer treatment, and the company also plans to apply for the same status in Australia.
In Europe, a rare disease is defined as one that occurs in less than 5 of every 10,000 people. Pancreatic cancer certainly meets the criteria in Europe and in the U.S., and the treatment has previously received the orphan drug designation in Europe. The company should receive the status with its U.S. and European applications, and if the biotech can, in fact, obtain both orphan drug designations, then Nuvilex should present even more value to Big Pharma.