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Pathways to Progress: How Orphan Drug Designation Sparks Biotech Transformation

NetworkNewsWire Editorial Coverage


New York, NY – September 20, 2023 – More than 30 million people in the United States suffer with a rare disease; most of them are receiving no current treatment. Supporting the development and evaluation of new treatments, particularly for rare diseases, is a key priority for the U.S. Food & Drug Administration. As part of that process, the agency can grant Orphan Drug Designation (ODD) to a drug or biological product being developed to prevent, diagnose or treat a rare disease or condition. This designation is intended to spark innovation among biotech companies that are developing treatments for these patient populations, which by definition affect fewer than 200,000 people in the U.S., by providing incentives such as tax credits, user-fee exemptions and up to seven years of market exclusivity after FDA approval. Last month, Genprex Inc. (NASDAQ: GNPX) (Profile) was granted FDA orphan drug designation for its REQORSA(R) immunogene therapy in development for the treatment of small cell lung cancer (SCLC). The Phase 1/2 clinical trial, expected to dose the first patient in the fourth quarter of 2023, uses a combination of REQORSA and Genentech Inc.’s Tecentriq(R) as maintenance therapy in patients with extensive stage small cell lung cancer (“ES-SCLC”) who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. Genprex joins other companies — including Roche Holding AG ADR (OTCQX: RHHBY), Jazz Pharmaceuticals PLC (NASDAQ: JAZZ), ALX Oncology Holdings Inc. (NASDAQ: ALXO) and Achilles Therapeutics PLC (NASDAQ: ACHL) — that have been granted ODD status or may seek ODD status as they work to develop treatments for rare diseases.


  • Four decades after the Orphan Drug Act was passed, hundreds of ‘orphan’ drugs have been approved for use.
  • Genprex’s REQORSA Immunogene Therapy has gained both Orphan Drug Designation and Fast Track Designation.
  • With recent ODD and FTD designations, Genprex is on the verge of commencing patient treatment in small cell lung cancer in Q4 2023.


Click here to view the custom infographic of the Genprex editorial.


Launching Point for Revolution


More than 30 million Americans struggle with rare diseases. A comprehensive study published by the National Library of Medicine (NLM) states that the 1983 passage of the Orphan Drug Act (ODA), which created the Orphan Drug Designation Program, represented a launching point for a rare-disease drug-development revolution for these patients.


“Drug development for rare diseases can be commercially risky, and prior to this legislation, only approximately two drugs per year had been approved by the U.S. Food and Drug Administration for rare diseases,” the report observed. “Now, four decades later, hundreds of ‘orphan’ drugs have been approved for use in the 7,000­–10,000 diseases and conditions that are considered rare.”


Specifically, the report noted that 6,340 orphan drug designations were granted, representing drug development for 1,079 rare diseases. “Additionally, 882 of those designations resulted in at least one FDA approval for use in 392 rare diseases,” the report continued. “Much of this development has been concentrated in oncology as seven of the top ten most designated and approved diseases were rare cancers. Based on our study, we can conclude that around 5% of rare diseases have an FDA-approved drug and up to 15% of rare diseases have at least one drug that has been developed and shown promise in their treatment, diagnosis or prevention. Funding of basic and translational science for rare-disease drug development should continue in order to bring therapies to the millions of affected patients who remain without treatment options.”


Bringing Hope


As the NLM article observed, there is a growing recognition of the potential of gene therapies to transform cancer care. Genprex (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, is among those companies leveraging this trend. The company is currently working in both the non-small cell lung cancer (NSCLC) and the small cell lung cancer (SCLC) space, where its REQORSA Immunogene Therapy (quratusugene ozeplasmid) has gained both Orphan Drug Designation and Fast Track Designation (FTD).


“We are excited to receive Orphan Drug Designation from the FDA for REQORSA for patients with SCLC,” said Rodney Varner, Genprex president, chair and CEO. “This FDA Orphan Drug Designation in combination with our recently received FDA Fast Track designation underscores the great need for better treatment options for patients with ES (extensive-stage)-SCLC and non-small cell lung cancer (NSCLC). We look forward to initiating the Acclaim-3 clinical trial expected in the fourth quarter of 2023 in order to bring hope of an effective new therapy to patients suffering with this life-limiting cancer.”


Meeting the SCLC Need


In the United States, lung cancer is the most lethal cancer in both men and women, with the American Cancer Society estimating that almost 130,000 people will die this year alone. SCLC comprises approximately 14% of all lung cancers. Limited stage SCLC (LS-SCLC) accounts for approximately 33% of all SCLC and is potentially curable with a combination of cytotoxic chemotherapy and radiation therapy. However, extensive stage (ES-SCLC), which comprises approximately 66% of all SCLC, is not considered curable. There is an unmet need for more effective maintenance therapy options for ES-SCLC, as well as for better combination treatments for SCLC overall.


This is the space Genprex is looking to fill with its REQORSA Immunogene Therapy. The company received the FTD approval for REQORSA Immunogene Therapy in combination with Genentech Inc.’s Tecentriq(R). Genentech is the U.S.-based unit of Swiss pharma giant Roche.


With ES-SCLC, cancer has spread from one lung to the other, or to other parts of the body. Genprex will be combining its drug with Genentech’s Tecentriq to focus on this type of cancer during the upcoming Acclaim-3 clinical trial. The phase 1/2 open-label, dose escalation and clinical response study will evaluate REQORSA in combination with Tecentriq in patients with ES-SCLC who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment.


Genprex has also been granted FTD approval for two other indications of REQORSA Immunogene Therapy: REQORSA in combination with Tagrisso for NSCLC in patients who have progressed after Tagrisso treatment, and REQORSA in combination with Keytruda for NSCLC in patients who have progressed after Keytruda treatment.


Transforming Lives, Redefining Treatments


Genprex operates in the dynamic fields of gene therapy, specifically targeting cancer and diabetes. With a focus on developing groundbreaking treatments for patients, the company’s market is characterized by the urgent need for innovative solutions. The cancer and diabetes sectors are vast and rife with unmet medical needs, offering significant opportunities for pioneering therapies that can transform lives and redefine treatment paradigms.


Genprex’s potential for growth is underscored by its promising pipeline of advanced gene therapies in development. REQORSA is the company’s lead candidate, which it hopes will revolutionize the treatment of lung cancers. With the recent ODD and FTD designations, along with a robust clinical-development program, Genprex is poised to make a significant impact in the market.


Elevating Innovation


Finding solutions in the rare disease sector can be challenging but certainly fulfilling as companies research, explore and strive to help those who need it the most. Add the incentives that come with an Orphan Drug Designation, and those efforts can become even more rewarding for these companies.


Roche Holding AG ADR (OTCQX: RHHBY), through Genentech, has received several Orphan Drug Designations. In addition to atezolizumab for the treatment of adult patients with ES-SCLC, Genentech most recently received Orphan Drug Designation for pralsetinib in the treatment of certain types of solid tumors. The ODD designation covers the use of pralsetinib in metastatic RET fusion-positive solid tumors. According to the terms, Roche has exclusive rights to commercialize the treatment outside the U.S. except for Greater China.


Jazz Pharmaceuticals PLC (NASDAQ: JAZZ), a global biopharmaceutical company dedicated to developing life-changing medicines for people with serious diseases, has also developed a drug for the SCLC market. Its Zepzelca(TM) (lurbinectedin), which was developed in collaboration with PharmaMar, was granted Orphan Drug Designation in 2018, and in 2020, it received accelerated FDA approval based on overall response rate (ORR) and duration of response.


ALX Oncology Holdings Inc. (NASDAQ: ALXO), a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, received ODD from both the FDA and the European Commission for its evorpacept, a next-generation CD47 blocker. The ODD indication includes both gastric cancer and gastroesophageal junction adenocarcinoma. “Receiving orphan drug designation from both the EC and the FDA represents a significant regulatory achievement for ALX Oncology and signifies the growing recognition of evorpacept as a potential new drug to improve clinical outcomes in patients with GC,” said ALX Oncology chief medical officer Sophia Randolph, MD., PhD.


Achilles Therapeutics PLC (NASDAQ: ACHL) is a biopharmaceutical company developing precision T cell therapies that target clonal neoantigens, or protein markers unique to each individual. Lead indications, including advanced NSCLC and recurrent metastatic melanoma, were selected for their high mutational burden, high level of T cell infiltration and high unmet medical need. Deaths from non-small cell lung cancer outnumber those resulting from breast cancer, colon cancer and prostate cancer combined, and there are limited approved treatment options for lung cancer and melanoma patients whose disease has not responded well to other therapies. In its March 25, 2021, SEC filing, the company noted that it “may in the future seek orphan drug designation for ATL001 and any future product candidates.”


For companies looking to help those in the underserved rare-disease space, an Orphan Drug Designation is the ideal pathway to progress. When a company such as Genprex, which truly is committed to innovation and patient-centric approaches, receives the designation, it becomes an even more distinctive differentiator. As investors explore opportunities in the biotech sector, Genprex stands out with its cutting-edge therapies, potential market disruption and dedication to transforming lives through innovative science.


For more information about Genprex Inc., please visit Genprex.


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