Fresh from receiving $16 million in Silicon Valley venture capital, Triangle drug startup Scioderm now has good news to talk about for its potential product lineup.

On Monday, Scioderm said the Food and Drug Administration had assigned “Breakthrough Therapy” designation to its lead product known as SD-101.

According to the FDA, only 10 of 40 applications for the designation have been awarded.

The new Breakthrough Therapy designation means expedited development and review are ahead, the company says. 

The company is preparing for clinical trials of an experimental drug addressing a rare genetic skin disease that has no cure or effective treatment.

The financing and new FDA status followed a review last month from the FDA that allowed the Raleigh company’s investigational new drug application for SD-101 to proceed.

SD-101 is a topical treatment that Scioderm is developing to treat Epidermolysis Bullosa, or EB. The rare genetic condition results in extremely fragile skin that blisters or tears at the slightest amount of friction. EB also affects internal organs and bodily systems.

Children who have EB have are sometimes called “butterfly children” because their skin is as fragile as the wings of a butterfly. Patients who have the most severe forms of EB experience scarring, disfigurement and disability. They usually die before the age of 30. The only treatment options are wound care, pain management and preventative bandaging.

“We are truly honored to have received Breakthrough Therapy designation for SD-101 and are pleased with the FDA’s decision to place our product in a category that may enable expedited development and review for patients with Epidermolysis Bullosa,” said Chief Executive Officer Robert Ryan.

“Given the recent important milestones for Scioderm, including the Series A closing along with this designation, we are looking forward to the opportunity to collaborate more closely with the FDA and potentially expedite the availability of an important new treatment option for patients with a disease that has no current effective treatment,” he added.

The Breakthrough Therapy Designation came about through the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA).

So what is the “Breakthrough Therapy” designation?

The FDA explains:

“Breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions.

“The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A breakthrough therapy designation conveys all of the fast track program features (see below for more details on fast track designation), as well as more intensive FDA guidance on an efficient drug development program.

“The FDA also has an organizational commitment to involve senior management in such guidance. Section 902 of FDASIA requires the following actions, as appropriate:

  • “holding meetings with the sponsor and the review team throughout the development of the drug
  • “providing timely advice to, and interactive communication with, the sponsor regarding the development of the drug to ensure that the development program to gather the nonclinical and clinical data necessary for approval is as efficient as practicable
  • “taking steps to ensure that the design of the clinical trials is as efficient as practicable, when scientifically appropriate, such as by minimizing the number of patients exposed to a potentially less efficacious treatment
  • “assigning a cross-disciplinary project lead for the FDA review team to facilitate an efficient review of the development program and to serve as a scientific liaison between the cross-discipline members of the review team (i.e., clinical, pharmacology-toxicology, chemistry, manufacturing and control (CMC), compliance) for coordinated internal interactions and communications with the sponsor through the review division’s Regulatory Health Project Manager
  • “involving senior managers and experienced review staff, as appropriate, in a collaborative, cross-disciplinary review”

(More information can be read online.)

Last week, two Silicon Valley-based venture capital firms lead a $16 million Series A round of financing. Leading the round was Morgenthaler Ventures. Technology Partners also participated in the deal.

Last month, Scioderm disclosed raising $1 million.