PALO ALTO, Calif. - BridgeBio Pharma, Inc. (NASDAQ: NASDAQ:BBIO) announced that the U.S. Food and Drug Administration (FDA) has awarded Breakthrough Therapy Designation to its oral drug candidate infigratinib, currently under development for treating children with achondroplasia, a form of dwarfism. This status is intended to expedite the development and review process for drugs that show potential to offer substantial improvements over existing therapies on significant clinical endpoints.
The designation was granted based on results from the PROPEL 2 clinical trial, which indicated that infigratinib led to a statistically significant and sustained increase in annualized height velocity (AHV) in children. Specifically, Cohort 5 of the trial demonstrated a mean change from baseline in AHV of +2.51 cm/year at 12 months and +2.50 cm/year at 18 months with a p-value of 0.0015. Additionally, improvements in body proportionality were observed at the 18-month mark, with a statistically significant p-value of 0.001.
Infigratinib, if approved, could be the first oral treatment option for this patient population. Achondroplasia not only affects height but can also impact health-related quality of life and functionality. The advocacy group Growing Stronger, which supports research and resources for individuals with achondroplasia, has expressed optimism about the FDA's recognition of the drug's potential.
BridgeBio's ongoing global Phase 3 registrational study, PROPEL 3, is on track with its enrollment, with completion expected by the end of the year. The company has also secured Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from the FDA for infigratinib, which could lead to a Priority Review Voucher if the drug is approved.
The pharmaceutical company, founded in 2015, is committed to addressing unmet needs in genetic diseases through its diverse pipeline of development programs. BridgeBio's focus extends beyond achondroplasia to include hypochondroplasia and other skeletal dysplasias. This announcement is based on a press release statement from BridgeBio Pharma, Inc.
In other recent news, BridgeBio Pharma's gene therapy BBP-812 has earned expedited FDA review after being granted Regenerative Medicine Advanced Therapy (RMAT) designation. The therapy, developed for Canavan disease, demonstrated promising results in the CANaspire Phase 1/2 trial, leading to functional improvements and reductions in disease biomarkers in all assessed patients. BridgeBio also announced the discontinuation of its BBP-631 gene therapy program, a move projected to save over $50 million in research and development.
Several analyst firms, including H.C. Wainwright, Piper Sandler, Oppenheimer, Mizuho, and TD Cowen, have maintained positive ratings for BridgeBio. These endorsements follow the recent developments in the company's investigational drug, acoramidis, and the expected release of phase 3 data for encaleret and infigratinib.
BridgeBio has also formed a joint venture named GondolaBio, backed by a $300 million investment from a consortium of investors, aiming to expedite the development of new therapies. These are all recent developments in the company's journey towards advancing treatments for genetic diseases.
InvestingPro Insights
As BridgeBio Pharma, Inc. (NASDAQ: BBIO) continues to make strides with its promising drug infigratinib, the financial metrics and analyst outlooks from InvestingPro indicate a dynamic picture for the company. According to recent data, BridgeBio has a substantial market capitalization of $4.92 billion, which reflects investor confidence in the company's potential despite the lack of current profitability.
One of the key InvestingPro Tips highlights that analysts are expecting sales growth for BridgeBio in the current year, aligning with the positive developments in the company's clinical trials. This anticipated growth is underscored by a staggering revenue growth of 3761.22% over the last twelve months as of Q2 2024, signaling a significant expansion in BridgeBio's financial scale. Moreover, two analysts have revised their earnings projections upwards for the upcoming period, suggesting that the market is optimistic about the company's future performance.
However, it's important to note that the company's stock has recently taken a hit, with a one-week price total return showing a decline of 12.7%. This volatility may be reflective of the high-risk, high-reward nature of investing in biotech firms, especially those like BridgeBio that are in the process of seeking regulatory approval for groundbreaking therapies. The company does not pay dividends, which is typical for growth-focused biotech companies reinvesting earnings into research and development.
For readers interested in a deeper dive into BridgeBio's financial health and future prospects, InvestingPro offers additional tips and insights. There are currently 7 additional InvestingPro Tips available for BridgeBio, providing a more comprehensive view of the company's investment potential.
With a robust pipeline and the potential for infigratinib to become the first oral treatment for achondroplasia, BridgeBio's journey is one to watch closely. For further details and expert analysis, investors can visit InvestingPro at: https://www.investing.com/pro/BBIO
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