Relief’s Inhaled Therapy RLF-100 Earns FDA’s Orphan Drug Status

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by Marta Figueiredo PhD |

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The U.S. Food and Drug Administration has granted orphan drug designation to RLF-100 (aviptadil), Relief Therapeutics’ investigational inhaled therapy for sarcoidosis.

Such status is given to treatment candidates with the potential to be safe and effective in rare diseases — defined in the U.S. as those affecting fewer than 200,000 people — that have no approved treatments, or in cases in which the potential therapy shows significant benefit over existing treatments.

The designation is meant to accelerate RLF-100’s clinical development and review by providing regulatory support and financial benefits, as well as seven years of marketing exclusivity in the U.S. if FDA approval is granted.

“Receipt of our third Orphan Drug Designation is another important milestone for the company, as it underscores the potential strength of our pipeline and the high need for better treatments for rare diseases such as sarcoidosis,” Raghuram (Ram) Selvaraju, PhD, chairman of Relief’s board of directors, said in a press release.

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Sarcoidosis is a highly variable inflammatory disease that can lead to the formation of small abnormal lumps of cells — nodules called granulomas — in virtually any organ in the body, but most commonly the lungs.

RLF-100 is an artificial form of vasoactive intestinal peptide, a molecule naturally produced throughout the body and mainly concentrated in the lungs, that has potent anti-inflammatory, immunoregulatory, and anti-viral properties.

In January, Relief reached a deal with AdVita Lifescience, the original developer of RLF-100’s inhaled formulation, to acquire the company and all of its assets.

Prior research showed that RLF-100’s inhaled formulation reduces lung inflammation and protects lung cells from injury. Its use was found to preserve the production of lung surfactant, which keeps lung tissue moist and lubricated. That, in turn, helps lung tissue maintain its elasticity, reducing the amount of work required to expand and contract it.

Notably, a previous proof-of-concept Phase 2 clinical trial, called Avisarco II (2017-004219-37), showed the therapy was generally well-tolerated and safe. That trial had evaluated the safety and effectiveness of inhaled RLF-100 in 20 adults with pulmonary sarcoidosis associated with cough.

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Treatment with RLF-100 led to clinically significant suppression of inflammatory processes in the lung and a reduction in sarcoidosis-relevant biomarkers. RLF-100 also was found to lessen patients’ dry cough and shortness of breath during exercise.

These findings suggested that RLF-100 may be a potentially safe and effective treatment for easing the symptoms of pulmonary sarcoidosis. Relief is planning to advance the clinical development of RLF-100 for this indication later this year.

“The timing of this newest Orphan Drug Designation comes on the heels of our just closed acquisition of AdVita Lifescience GmbH and supplements those we have for our drug candidates for [epidermolysis bullosa] and [phenylketonuria], which we added to our pipeline through our recent acquisition of APR Applied Pharma Research SA, consistent with our strategy to become a fully integrated diversified commercial-stage pharmaceutical company,” Selvaraju said.

RLF-100’s inhaled and into-the-vein formulations also are being tested in late-stage clinical trials in the U.S. and Europe for the treatment of severe COVID-19 respiratory complications.