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FDA Accepts BridgeBio's Filing for Rare Genetic Disease Drug
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Key Takeaways
BridgeBio's filing for encaleret in ADH1 was accepted by the FDA, with a decision due by May 8, 2027.
BBIO's filing is backed by phase III data showing restored blood and urine calcium and PTH production.
BridgeBio says encaleret could expand its portfolio beyond Attruby alongside BBP-418 and infigratinib.
BridgeBio Pharma (BBIO - Free Report) announced that the FDA has accepted its regulatory filing seeking approval for the investigational oral candidate encaleret to treat individuals living with a rare genetic endocrine disorder called autosomal dominant hypocalcemia type 1 (ADH1).
A final decision is expected by May 8, 2027. If approved, encaleret would become the first FDA-approved therapy specifically indicated for ADH1, offering a disease-targeted treatment for a condition that is currently managed with calcium and active vitamin D supplementation rather than therapies that address its underlying cause.
The FDA also notified BridgeBio that it is not currently planning to hold an advisory committee meeting, suggesting that the agency does not presently see the need for external expert review of the application. While this is generally viewed as a positive procedural development, it should not be interpreted as an indication of the FDA's ultimate approval decision.
The filing is supported by results from the phase III CALIBRATE study, which showed that encaleret led to the simultaneous restoration of blood and urine calcium, as well as the restoration of physiologic parathyroid hormone (PTH) production. Per BridgeBio, the findings support the drug’s potential as a disease-modifying therapy by targeting the underlying genetic cause of ADH1.
BBIO Stock’s Price Performance
Year to date, the company’s shares have gained 8% against the industry’s 5% fall.
Image Source: Zacks Investment Research
BridgeBio Takes a Step Closer to Becoming a Multi-Product Company
The FDA's acceptance of encaleret marks another regulatory milestone for BridgeBio as it continues to expand its product portfolio beyond Attruby, which is currently its only marketed product. The drug is approved for the treatment of adults with transthyretin amyloid cardiomyopathy (ATTR-CM).
However, the company has several late-stage candidates that could significantly diversify its revenue base over the next 12 months. Encaleret is one of three near-term commercialization opportunities, alongside BBP-418 and infigratinib.
A filing for BBP-418 is already under FDA review for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), with a final decision expected by Nov. 27, 2026. On the other hand, BridgeBio is on track to submit a filing to the agency for infigratinib as a potential treatment for achondroplasia in the third quarter of 2026.
Together with encaleret, BBP-418 and infigratinib could significantly diversify BridgeBio's revenue base. If approved, these candidates would transform the company from a single-product business into a diversified rare disease commercial player, reducing its dependence on Attruby as its primary growth driver.
BBIO’s Zacks Rank
BridgeBio currently carries a Zacks Rank #3 (Hold).
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share (EPS) have increased from $2.97 to $3.02. Over the same period, EPS estimates for 2027 have also increased from $4.81 to $4.92. LQDA shares have skyrocketed more than 150% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed once, with the average surprise being 54.40%.
Over the past 60 days, estimates for Harmony Biosciences’ 2026 EPS have increased from $3.20 to $3.30. Over the same period, EPS estimates for 2027 have risen from $3.64 to $3.87. HRMY shares have lost nearly 7% year to date.
Harmony Biosciences’ earnings missed estimates in each of the trailing four quarters, with the average negative surprise being 25.16%.
View the Zacks #1 Rank List
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FDA Accepts BridgeBio's Filing for Rare Genetic Disease Drug
Key Takeaways
BridgeBio Pharma (BBIO - Free Report) announced that the FDA has accepted its regulatory filing seeking approval for the investigational oral candidate encaleret to treat individuals living with a rare genetic endocrine disorder called autosomal dominant hypocalcemia type 1 (ADH1).
A final decision is expected by May 8, 2027. If approved, encaleret would become the first FDA-approved therapy specifically indicated for ADH1, offering a disease-targeted treatment for a condition that is currently managed with calcium and active vitamin D supplementation rather than therapies that address its underlying cause.
The FDA also notified BridgeBio that it is not currently planning to hold an advisory committee meeting, suggesting that the agency does not presently see the need for external expert review of the application. While this is generally viewed as a positive procedural development, it should not be interpreted as an indication of the FDA's ultimate approval decision.
The filing is supported by results from the phase III CALIBRATE study, which showed that encaleret led to the simultaneous restoration of blood and urine calcium, as well as the restoration of physiologic parathyroid hormone (PTH) production. Per BridgeBio, the findings support the drug’s potential as a disease-modifying therapy by targeting the underlying genetic cause of ADH1.
BBIO Stock’s Price Performance
Year to date, the company’s shares have gained 8% against the industry’s 5% fall.
Image Source: Zacks Investment Research
BridgeBio Takes a Step Closer to Becoming a Multi-Product Company
The FDA's acceptance of encaleret marks another regulatory milestone for BridgeBio as it continues to expand its product portfolio beyond Attruby, which is currently its only marketed product. The drug is approved for the treatment of adults with transthyretin amyloid cardiomyopathy (ATTR-CM).
However, the company has several late-stage candidates that could significantly diversify its revenue base over the next 12 months. Encaleret is one of three near-term commercialization opportunities, alongside BBP-418 and infigratinib.
A filing for BBP-418 is already under FDA review for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), with a final decision expected by Nov. 27, 2026. On the other hand, BridgeBio is on track to submit a filing to the agency for infigratinib as a potential treatment for achondroplasia in the third quarter of 2026.
Together with encaleret, BBP-418 and infigratinib could significantly diversify BridgeBio's revenue base. If approved, these candidates would transform the company from a single-product business into a diversified rare disease commercial player, reducing its dependence on Attruby as its primary growth driver.
BBIO’s Zacks Rank
BridgeBio currently carries a Zacks Rank #3 (Hold).
BridgeBio Pharma, Inc. Price
BridgeBio Pharma, Inc. price | BridgeBio Pharma, Inc. Quote
Our Key Picks Among Biotech Stocks
Some better-ranked stocks from the sector are Liquidia Corporation (LQDA - Free Report) and Harmony Biosciences (HRMY - Free Report) , sporting a Zacks Rank #1 (Strong Buy) each at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share (EPS) have increased from $2.97 to $3.02. Over the same period, EPS estimates for 2027 have also increased from $4.81 to $4.92. LQDA shares have skyrocketed more than 150% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed once, with the average surprise being 54.40%.
Over the past 60 days, estimates for Harmony Biosciences’ 2026 EPS have increased from $3.20 to $3.30. Over the same period, EPS estimates for 2027 have risen from $3.64 to $3.87. HRMY shares have lost nearly 7% year to date.
Harmony Biosciences’ earnings missed estimates in each of the trailing four quarters, with the average negative surprise being 25.16%.