FDA Approves First Immunotherapy for Rare Respiratory Disorder, Marking Significant Milestone in HPV-Related Treatment
The U.S. Food and Drug Administration (FDA) has granted approval to Precigen's Papzimeos (zopapogene imadenovec-drba), the first-ever immunotherapy treatment for recurrent respiratory papillomatosis (RRP). This groundbreaking approval marks a significant advancement in the treatment of this rare, chronic disease caused by human papillomavirus (HPV) types 6 and 11.
A New Era in RRP Treatment
Papzimeos, a non-replicating adenoviral vector-based immunotherapy, offers hope to patients who have long endured repeated surgical interventions to manage their condition. The treatment works by triggering an immune response against HPV-infected cells, potentially eliminating the need for frequent surgeries.
Dr. Helen Sabzevari, CEO of Precigen, emphasized the historic nature of this approval, stating, "It cannot be underscored enough what an historic moment it is with the approval of Papzimeos for this patient population. It has changed the paradigm for treatment of these patients."
The FDA's decision came nearly two weeks ahead of the target date, with Dr. Vinay Prasad, Director of the FDA's Center for Biologics Evaluation and Research (CBER), noting that this approval demonstrates the agency's flexibility in evaluating treatments for rare diseases.
Clinical Efficacy and Patient Impact
The approval was supported by impressive clinical trial results involving adult RRP patients requiring at least three surgeries per year. Key findings include:
- 51% of patients achieved a complete response, defined as not needing surgical intervention for 12 months post-treatment.
- 15 out of 18 complete responders maintained their response through two years of follow-up.
- Patients averaged only 0.5 surgeries in the year following treatment, a significant reduction from their pre-treatment frequency.
These results offer a promising outlook for RRP patients like Kim McClellan, President of the RRP Foundation, who has undergone over 250 surgeries since her diagnosis at age 5.
Market Implications and Commercial Strategy
Precigen's stock saw a significant boost following the announcement, with shares rising by approximately 44-50% in pre-market trading. The company has identified about 27,000 adult RRP patients in the U.S., suggesting a substantial market for Papzimeos.
To support the launch, Precigen has enlisted the expertise of Phil Tennant, a pharmaceutical industry veteran, as chief commercial officer. The company has also partnered with Chicago-based Eversana for commercialization support.
As the pharmaceutical industry continues to advance treatments for rare diseases, Papzimeos stands out as a beacon of progress, offering new hope to patients who have long suffered from this challenging condition.
References
- Precigen Wins First FDA Immunotherapy Nod for Recurrent Respiratory Papillomatosis
Papzimeos is the first immunotherapy approved for recurrent respiratory papillomatosis, a rare lung disorder involving the development of benign tumors in the airways.
- Precigen scores FDA nod for first-ever treatment for HPV-related disorder
The FDA has approved a treatment for recurrent respiratory papillomatosis for the first time. The agency has signed off on Precigen’s Papzimeos, a non-replicating adenoviral vector-based immunotherapy, which can eliminate the need for repeated surgical interventions to remove benign tumors from the respiratory tract.
Explore Further
What are the specific mechanisms that Papzimeos employs to trigger an immune response against HPV-infected cells?
How does the efficacy of Papzimeos compare to existing treatments for recurrent respiratory papillomatosis in terms of reducing surgical interventions?
What role did Dr. Vinay Prasad and the FDA's Center for Biologics Evaluation and Research play in the expedited approval process of Papzimeos?
Given Precigen's partnership with Eversana, what are the anticipated strategies for commercializing Papzimeos in the U.S. healthcare market?
How does the financial performance of Precigen following the Papzimeos approval compare to other biotech companies pursuing therapies for rare diseases?