Orphan drug designation granted in US and EU for systemic sclerosis treatment

AnaMar, a clinical-stage biotech company developing first in class anti-fibrotic 5-HT2B receptor antagonists, has been granted orphan drug designation (ODD) by the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) for its lead clinical candidate, AM1476 for the treatment of systemic sclerosis (SSc).

AM1476 is a selective peripheral-acting 5-HT2B receptor small molecule antagonist that can be delivered orally. It has demonstrated efficacy in in vitro and in vivo models of fibrosis, as well as favourable safety and pharmacokinetic profiles in Phase I clinical studies. AM1476 offers a unique dual-action approach to treat both skin and lung manifestations of systemic sclerosis.

Systemic sclerosis is a chronic, progressive, autoimmune disease characterized by inflammation and fibrosis, i.e. uncontrolled scar tissue formation, in skin and various internal organs. Skin fibrosis is the distinguishing hallmark of SSc, associated with significant disability. The degree of skin fibrosis provides insight about progression of the disease.

Interstitial lung disease (ILD) is a common and early manifestation of SSc. Around 100,000 people in the EU are affected by SSc and up to 80% of these may develop ILD. ILD causes progressive lung scaring, known as fibrosis, which leads to increasing, chronic, breathing problems and organ dysfunction, and can lead to death. There are currently no treatments on the market that effectively stop or reverse scarring in both skin and lung tissue.

AnaMar’s Chief Executive Officer, Dr. Ulf Ljungberg, said: “We are delighted with the FDA’s and EMA’s decisions to grant orphan drug designation to AM1476 for SSc. This is a significant milestone and underscores the significant unmet need for novel medicines to prevent, heal and slow organ scarring from fibrotic diseases, which are often progressive and can have a poor prognosis.

“There is great potential in AM1476 as a unique dual-action approach to treat skin and lung manifestations of systemic sclerosis, especially as it represses both macrophage and fibroblast activity, whilst minimizing side effects and interactions with other medicines. We are now planning for Phase II clinical trials and look forward to commercialising our product with a pharma partner to bring better treatment options to patients with fibrosis.”

The company has designed a Phase II study to evaluate the treatment effects in SSc-ILD with a proposed dosing regimen for 60 patients in a double-blinded, placebo-controlled randomized trial over 12 months with lung function and skin thickness as efficacy readouts. Drawing on its heritage in diagnostics, the company is also developing biomarkers and gene signatures to identify patients most likely to respond to treatment and ensure the best outcomes for patients with fibrosis.

The FDA and EMA grant orphan status to products intended to treat, diagnose, or prevent a life-threatening rare disease or condition that affects fewer than five in 10,000 people in Europe, or under 200,000 people in the US, and with either no currently approved method of diagnosis, prevention, or treatment, or with significant benefit to those affected by the disease. Orphan drug designation provides certain benefits, including the potential for extensive marketing exclusivity following regulatory approval, reduction in regulatory fees and, in the case of EU, a centralized approval process.

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