Gesynta Pharma’s drug candidate GS-248 granted Orphan Drug Designation in
US by the FDA for the treatment of systemic sclerosis
Stockholm, Sweden, 27 April 2022 – Gesynta Pharma AB today announces that the U.S. Food and
Drug Administration (FDA) has granted Orphan Drug Designation to the company’s drug candidate
GS-248 for the treatment of systemic sclerosis. GS-248 is currently being evaluated in a Phase II
clinical trial as a treatment for Raynaud’s phenomenon secondary to systemic sclerosis.
Orphan Drug Designation is intended to encourage the development of drugs for the treatment of
rare diseases. FDA may grant orphan drug designation to drugs or biological products intended to
treat diseases that affect less than 200,000 people in the United States. During the drug
development stage, the designation provides certain benefits such as tax credits for clinical trials
conducted in the US, and exemption from FDA user fees for marketing approval. At a later stage,
orphan drug designation provides the opportunity for marketing approval as an Orphan Drug, and up
to seven years of market exclusivity.
As part of its overall strategy, Gesynta Pharma has worked diligently to prepare for upcoming clinical
studies in the US, and the company has conducted several successful advisory meetings with the FDA
over the past year. Gesynta Pharma also has an open IND (Investigational New Drug Application) for
GS-248.
“The orphan drug designation granted to our drug candidate GS-248 for the treatment of systemic
sclerosis provides an opportunity for extended market exclusivity, which is a valuable complement to
its strong patent protection. Strengthened by this positive news and our constructive dialogue with
the FDA, we continue working towards the goal of being able to offer an effective and safe treatment
for patients with systemic sclerosis”, says Patric Stenberg, CEO of Gesynta Pharma.
Systemic sclerosis (also known as scleroderma) is a chronic, autoimmune disease that affects, among
other things, the body’s smallest vessels, the microvasculature, and is associated with great medical
needs due to the current lack of safe and effective medicines. Already at an early stage of the
disease, patients suffer from episodes of impaired blood flow in the fingers and toes, Raynaud’s
phenomenon, which in the course of the disease often lead to very painful, difficult-to-heal, and
frequently disabling ulcers. The lungs, kidneys and heart can also be seriously damaged due to
inflammation and damage to the microvasculature. GS-248 has a unique and promising mechanism
of action with the potential to help both patients with systemic sclerosis and patients with other
chronic inflammatory diseases.
For more information, please contact:
Patric Stenberg, CEO
Gesynta Pharma AB
Tel: + 46 (0)733 83 66 70
E-mail: [email protected]