News

Video

Setting Precedents for Rare Disease Therapies

Helen Sabzevari, PhD, president and CEO of Precigen, reflects on how Papzimeos sets an important precedent for rare disease therapies, with its broad FDA label for all adult patients with recurrent respiratory papillomatosis (RRP) highlighting the potential for earlier intervention to prevent irreversible damage and establish new standards of care.

Pharmaceutical Executive: What precedent do you think Papzimeos sets for future therapies in rare diseases?
Helen Sabzevari: So this led to papzimeos being the standard of care, the first and only FDA approved drug for RRP, and the label is for all adult RRP patients, which is a broad label. And I think this is very important, because now any of the adults diagnosed with the RRP, they are eligible to receive the papzimeos starting and the reason that this is important for this patient population, studies coming out of John Hopkins have shown that these patients, by the time they get to the fifth surgery, they already have irreversible damage to their vocal cords and to their trachea. So definitely, it becomes not only important for patients that they have severe cases, but also even the patients that immediately start being diagnosed with the RRP, because, for sure, the physicians, they do not want these patients to get to that level of damage that they cannot reverse it back. And I think that's the strength of this drug, and also the importance of having a label that now all adult or RP patients can be treated with this.

Newsletter

Lead with insight with the Pharmaceutical Executive newsletter, featuring strategic analysis, leadership trends, and market intelligence for biopharma decision-makers.

Related Videos
Pharmaceutical Executive
Helen Sabzevari
Helen Sabzevari
© 2025 MJH Life Sciences

All rights reserved.