View Archives

How the 21st Century Cures Act Impacts InVivo – December 19, 2016

The 21st Century Cures Act passed the House and the Senate, and President Obama signed it into law on December 13. The Act provides immediate benefits for both patients and companies developing therapies for patients with orphan diseases or conditions. InVivo Therapeutics is studying the Neuro-Spinal Scaffold™ in patients with complete paralysis (no motor or sensory function below the level of injury) as a result of an acute spinal cord injury. We are utilizing the Humanitarian Device Exemption (HDE) pathway through the FDA, which previously capped the eligible population of patients to be treated at 4,000 annually. At 4,000 patients per year, InVivo was confined to taking advantage of the HDE pathway only in patients with complete paralysis. Under the 21st Century Cures Act, that cap is lifted to 8,000 patients per year, which may allow InVivo to take advantage of the HDE pathway for many patients with incomplete (some motor or sensory function) paralysis as well. To better understand the immediate implications to our clinical development portfolio, it’s helpful to describe the key differences between the HDE pathway we’ve been following with the INSPIRE study and the Premarket Approval (PMA) pathway we previously would have had to follow to address incomplete SCI.

The FDA states that the “PMA is the most stringent type of device marketing application required by FDA.”1 To obtain approval through the PMA pathway, a sponsor has to demonstrate “safety and effectiveness” usually by conducting an appropriately powered pivotal study to demonstrate the investigational product is statistically superior to a control. This is a suitable way of evaluating investigational therapies if the disease has a large prevalence or incidence, because such trials usually involve hundreds of people. The challenges associated with running large trials with very small patient populations was one of the drivers behind the creation of the HDE pathway. The bar for approval set by the HDE pathway is “safety and probable benefit,” which allows for smaller trials and a more streamlined path to approval.

InVivo is currently exploring the best regulatory strategy for expanding our HUD population. If an expanded incomplete SCI population is approved, we would plan to study incomplete SCI patients in a new study (following the INSPIRE study) designed to support HDE approval.

Given the early compelling data from the INSPIRE pivotal study, the potential to address patients with incomplete paralysis via the HDE regulatory pathway is extremely appealing.  There are no approved therapies for spinal cord injury today, representing an enormous unmet medical need. InVivo is working every day to change that, and this legislation may allow for a more rapid path to expanding the clinical and commercial potential of the Neuro-Spinal Scaffold.

Mark Perrin, CEO & Chairman
December 19, 2016

References

  1. http://www.fda.gov/medicaldevices/deviceregulationandguidance/howtomarketyourdevice/premarketsubmissions/premarketapprovalpma/

Safe Harbor Statement

Any statements contained in this CEO Perspective that do not describe historical facts may constitute forward-looking statements within the meaning of the federal securities laws. These statements can be identified by words such as “believe,” “anticipate,” “intend,” “estimate,” “will,” “may,” “should,” “expect,” “designed to,” “potentially,” and similar expressions, and include statements regarding the safety and effectiveness of the Neuro-Spinal Scaffold; the company’s ability to commercialize its products; the company’s ability to develop, market and sell products based on its technology; the expected benefits and efficacy of the company’s products and technology in connection with the treatment of spinal cord injuries; the availability of substantial additional funding for the company to continue its operations and to conduct research and development, clinical studies and future product commercialization; and other risks associated with the company’s business, research, product development, regulatory approval, marketing and distribution plans and strategies identified and described in more detail in the company’s Annual Report on Form 10-K for the year ended December 31, 2015, and its other filings with the SEC, including the company’s Form 10-Qs and current reports on Form 8-K. The company does not undertake to update these forward-looking statements.

Archives

CEO's Perspective - October 13, 2016

Epidemiology and Market Opportunity Around the World - July 13, 2016

The INSPIRE Study - February 29, 2016

InVivo in 2016: Looking Ahead - December 17, 2015

Building Value in a Pre-commercialization Biotech Company: Importance of Increased Exposure Within the Scientific and Medical Communities - September 21, 2015

Corporate Financing - What are the options? - July 29, 2015

Social Media: The New Frontier - June 3, 2015
What NASDAQ has to Offer - April 16, 2015

Reverse Splits and Uplisting - March 24, 2015

This blog may contain forward-looking statements within the meaning of federal securities laws. Forward-looking statements can be identified by words such as “anticipates,” “believes,” “plans,” “will,” “intends,” “expects,” and similar references to the future, and include statements InVivo may make regarding its product development strategy, business prospects, and operational milestones. Readers should not unduly rely on these forward-looking statements, which are not a guarantee of future performance and are subject to a number of risks and uncertainties, many of which are outside InVivo’s control, which could cause its actual business and operating results to differ. For more information on the many factors that can result in actual performance differing from these forward-looking statements, please see the risk factors detailed in InVivo’s SEC filings. InVivo does not undertake to update these forward-looking statements.