ReAlta receives Orphan Drug Designation and Fast Track Designation from the FDA for GvHD Therapy
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation and fast-track designation to ReAlta Life Sciences’ lead product candidate pegtarazimod (RLS-0071) for the treatment of steroid-refractory acute graft-versus-host disease (aGvHD).
These approvals come as the company continues its open-label Phase II AURORA trial (NCT06343792) of pegtarazimod in this indication. The FDA approvals provide ReAlta with seven years of post-approval market exclusivity for pegtarazimod and accelerate the review process.
AV hypertrophy is an often fatal complication after hematopoietic stem cell transplantation in which the donor’s immune cells attack the recipient’s tissue. The standard treatment is steroid therapy, but patients who do not respond to steroid therapy have limited further treatment options and a high risk of death. There are an estimated 4,000 such patients in the US, EU and Japan.
Pegtarazimod is based on ReAlta’s EPICC peptide platform. The technology is based on the human astrovirus HAstV-1, which causes non-inflammatory, self-limiting gastroenteritis by inhibiting components of the immune system.
Dr. Kenji Cunnion, chief medical officer at ReAlta, said the FDA’s decision “underscores the significant potential of RLS-0071 and its novel dual mechanism of action to address critical unmet needs of patients with this life-threatening disease.”
If successfully developed and approved, pegtarazimod will be able to compete in a market that includes Johnson & Johnson’s Imbruvica (ibrutinib), Novartis’ Jakavi or Jakafi (ruxolitinib phosphate) and Bristol Myers Squibb’s Orencia (abatacept). These therapies are forecast by GlobalData to generate annual sales of $2.56 billion, $2.25 billion and $3.44 billion, respectively, by 2030.
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Virginia-based ReAlta is currently investigating the use of its lead therapy candidate in a variety of indications, thanks in part to $20 million raised in a Series A funding round in 2021. In addition to the AURORA trial, the company is conducting Phase II trials to determine the efficacy of pegtarazimod in neonates with hypoxic-ischemic encephalopathy (NCT05778188) and in patients with acute exacerbations of chronic obstructive pulmonary disease (NCT06175065).
ReAlta also completed a Phase I study to investigate the therapeutic use of RLS-0071 for asthma (NCT05351671) and conducted a Phase I study to investigate the use of the drug in Covid-19-related acute respiratory distress syndrome (NCT04574869) before the study was terminated due to the “low likelihood of enrolling patients before the study drug expires,” according to ClinicalTrials.govEarlier this year, the company collaborated with the US National Institute of Allergy and Infectious Diseases (NIAID) to evaluate the use of the drug in acute radiation syndrome.
