What Happened This Week in the World of Pharmacy?

1. A New Drug Class for NASH?^1,2

Nonalcoholic fatty liver disease (NAFLD) is one of the most common causes of chronic liver disease and one of the most common liver disorders in the United States. Nonalcoholic steatohepatitis (NASH) is serious form of NAFLD and a major cause of cirrhosis. Currently, there are more than 3 million new US cases of NASH per year and no available treatment.

By 2020, the potential market for this disease is estimated to be $1.6 billion globally. There are at least 20 global phase 2 clinical trials and almost every major pharmaceutical company is in the race to be the first one into the NASH market.

Just last week, Allergan purchased 2 NASH treatment developers, Tobira and Akarna, for $1.7 billion and $50 million, respectively. We’ll likely see a new drug class in the upcoming years with the potential to create blockbuster sales like the hepatitis C medications.

2. Another EpiPen Scandal³

Mylan has been in the hot seat recently due to the EpiPen’s price hike, but the scandal doesn’t stop there. Apparently, Mylan has been misclassifying the medication under the Medicaid Drug Rebate Program for years. Instead of classifying EpiPen as a brand-name medication, Mylan classified it as a generic.

Under this program, drug manufacturers can get their products covered by Medicaid if they offer rebates to the government to offset costs. Drug manufacturers are required to offer a 13% rebate for generic medications and a 23.1% rebate for brand-name medications. They’re also required to include an inflation rebate any time the brand-name medication’s price increases at a rate higher than the yearly inflation rate.

The Centers for Medicare and Medicaid Services has reached out to Mylan multiple times for their incorrect classification, but Mylan hasn’t taken any action to change it. This means the government has been overpaying for the EpiPen for years. It was reported that Minnesota’s Medicaid program overpaid $4.3 million this year alone.

3. Accelerated Approval Granted to First Duchenne Muscular Dystrophy (DMD) Drug

DMD is a rare genetic disorder affecting 1 in 3600 male infants worldwide. It’s caused by a mutation in the gene for dystrophin, a protein that maintains the muscle fiber cell membrane. This life-threating disease causes progressive muscle deterioration and weakness and has an average life expectancy of 26 years.

There are currently no available treatments for this disease, but the FDA just approved Sarepta’s eteplirsen (Exondys 51), the first-ever drug approved to treat DMD, under an accelerated pathway. The drug’s intended for patients with a mutation of the dystrophin gene amenable to exon 51 skipping, which accounts for 13% of DMD patients.

Exondys 51 demonstrated that some patients had increased levels of dystrophin in their skeletal muscles. However, its clinical benefit of improved motor function hasn’t been established. The FDA is requiring Sarepta to conduct an additional 2-year clinical trial to confirm this claim, and if it fails to demonstrate this clinical benefit, the FDA will withdraw its approval.

4. Getting Closer to a Zika Vaccine⁴

Sanofi just received $43 million in funding from the US Biomedical Advanced Research and Development Authority (BARDA) for its candidate vaccine’s phase 2 clinical trials. BARDA could provide an additional $130 million if the phase 2 clinical trials are successful. Sanofi is expected to start its midstage trials in the first half of 2018.

5. New Dengue Fever Vaccine in Development⁵

Dengue fever is a serious disease affecting more than 110 countries worldwide. India accounts for approximately 50% of the global population at risk for this disease, and the medical care and indirect economic costs for those with dengue fever is estimated at over $1 billion.

Sanofi’s Dengvaxia is currently the only dengue fever vaccine available. However, it hasn’t been approved for use in India until Sanofi performs clinical trials on the local population.

In the meantime, Sun Pharma, India’s largest pharmaceutical company, has joined the race to develop a dengue fever vaccine. Earlier this week, Sun Pharma partnered with the New Delhi lab of the International Centre for Genetic Engineering and Biotechnology to develop its candidate vaccine, DSV4, which is designed to protect against all 4 subtypes of the dengue virus.

References

• McKee K. Nonalcoholic steatohepatitis: what’s the market opportunity and who are the major players? seekingalpha.com/article/4009113-nonalcoholic-steatohepatitis-market-opportunity-major-players. Published September 29, 2016.
• Nonalcoholic steatohepatitis. NIH. niddk.nih.gov/health-information/health-topics/liver-disease/nonalcoholic-steatohepatitis/Pages/facts.aspx. Published May 2014.
• Mangan D. Mylan 'incorrectly' classified EpiPens, shortchanging government: regulators. cnbc.com/2016/09/28/mylan-incorrectly-classified-epipens-shortchanging-government-regulators.html. Published September 28, 2016.
• Russel A. Sanofi SA gets BARDA funding for Zika trial. marketexclusive.com/sanofi-sa-nysesny-gets-barda-funding-zika-trial/32826/. Published September 27, 2016.
• Taylor P. Chasing Sanofi's Dengvaxia, Sun Pharma licenses dengue fever vaccine from Indian biotech lab. fiercepharma.com/pharma-asia/chasing-sanofi-s-dengvaxia-sun-pharma-licenses-dengue-fever-vaccine-from-indian-biotech. Published September 28, 2016.