Specialty Drug Approvals in 2013

Mar 26, 2014
Specialty drugs once again dominated the list of drug approvals from the Food and Drug Administration in 2013, making up nearly 70% of drugs approved.
  • Cancer
  • Diabetes
  • Inflammatory Conditions
  • Hepatitis C
  • Mutiple Sclerosis

Specialty drugs once again dominated the list of drug approvals from the Food and Drug Administration in 2013; of the 28 therapeutic drugs approved, 19 of them – or nearly 70% – were specialty medications.

Specialty drugs have represented an increasing percentage of FDA approvals as pharmaceutical manufacturers focus on discovering and developing drugs to treat complex and chronic diseases such as cancer, rheumatoid arthritis, hepatitis C and multiple sclerosis. For the third consecutive year, specialty drugs have accounted for the majority of all FDA approval activity, compared to just 33% in 2008.

Oral Oncology Blockbusters, Breakthroughs

Eight new cancer drugs were approved in 2013. Several new oral formulas, as well as targeted therapies that require specific genetic testing before prescribing, result in more treatment options for various patients. Two examples are Tafinlar® and Mekinist®, which were approved to treat advanced melanoma.

Another example, Kadcycla®, is a new treatment for patients with HER2-positive breast cancer. With projected annual sales of more than $1 billion, it could potentially be a blockbuster drug.

In 2013, the FDA also approved the first drugs considered breakthrough therapies. Under FDA guidelines established in July 2012, drugs designated as a breakthrough therapy undergo an expedited development and review process. In order to achieve the designation as a breakthrough therapy, the drug must meet criteria that it is intended to treat a serious or life-threatening condition and preliminary clinical evidence shows that the drug may demonstrate substantial improvement over existing therapies.

GazyvaTM and ImbruvicaTM were each designated as breakthrough therapies and approved by the FDA in 2013. Gazyva, a new infused therapy for the treatment of chronic lymphocytic leukemia (CLL), is expected to be a blockbuster medication. Imbruvica is an oral medication approved to treat patients with mantle cell lymphoma. In February this year, Imbruvica received an expanded approval for previously treated CLL.

These are the first drugs to gain approval under the new expedited process. Its designation as a breakthrough therapy helped Imbruvica gain approval four months sooner than expected.

New Treatments for Hepatitis C

Two new oral drugs – OlysioTM and SovaldiTM – were approved in late 2013 to treat different genotypes of hepatitis C, which affects about 2.7 million Americans. Sovaldi, with its $84,000 cost for a course of therapy, is also expected to be a blockbuster medication. One of the ways Sovaldi was approved for use is with ribavirin to treat patients with genotype 2 or genotype 3 chronic hepatitis C, which are rare. It is designated as a breakthrough therapy when used as part of an all-oral regimen for hepatitis C.

Sovaldi is the first drug approved as part of an all-oral regimen for hepatitis C. Genotype 2 and genotype 3 represent less than 25% of all hepatitis C cases. But there are additional regimens currently going through the FDA approval process that will increase competition for genotype 1, the most common genotype, representing about 75% of cases. The standard of care for treating this disease state is rapidly evolving.

Traditional Blockbusters

While more of the development is trending toward specialty, the traditional market had some key approvals in 2013 as well – namely, two new treatments for Type 2 diabetes drugs. That means more treatment options and greater competition in this market. Unlike specialty medications, these drugs target a broader population and have the potential to have quite an impact.

As the most common form of the disease, Type 2 diabetes affects about 24 million people and accounts for more than 90% of diabetes cases diagnosed in the U.S. Two drugs to gain approval in 2013 – Nesina® and Invokana® – present new treatment options for these patients.

Invokana is the first in a new class of drugs for treating Type 2 diabetes. As an oral therapy drug, it helps the body to excrete excess glucose through the urine rather than absorbing it in the blood. As the disease progresses, patients may need multiple drug therapies to help control their diabetes, and this drug provides them with another treatment option.

In tomorrow’s post, we’ll share an overview and analysis of the drugs expected to gain FDA approval in 2014.

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