BridgeBio (NASDAQ: BBIO) files NDA for first potential LGMD2I/R9 treatment

Rhea-AI Filing Summary

BridgeBio Pharma submitted a New Drug Application to the FDA for oral BBP-418 to treat limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The filing is supported by the Phase 3 FORTIFY trial’s 12‑month interim analysis, which met all pre‑specified primary and secondary endpoints.

Interim data showed rapid, consistent treatment effects, with statistically significant and clinically meaningful improvements in ambulation and pulmonary function and a favorable safety profile. BridgeBio expects, if approved, a potential U.S. launch in late 2026 or early 2027 and notes BBP-418 could become the first approved therapy for LGMD2I/R9 and potentially any form of limb-girdle muscular dystrophy.

Insights

Biopharma & regulatory analyst

BridgeBio advances BBP-418 with an NDA backed by positive Phase 3 data, targeting first-in-class status in LGMD2I/R9.

BridgeBio Pharma has submitted an NDA for BBP-418 in LGMD2I/R9, anchored by the Phase 3 FORTIFY 12‑month interim analysis meeting all primary and secondary endpoints. The company highlights statistically significant, clinically meaningful gains in ambulation and pulmonary function alongside a favorable safety profile.

BBP-418 already holds Orphan Drug, Fast Track and Rare Pediatric Disease designations in the U.S., plus Orphan Drug status in Europe. These designations, together with unmet need in LGMD2I/R9, mean the NDA may be eligible for Priority Review, and approval could generate a Priority Review Voucher.

If approved, BBP-418 would be the first and only therapy for LGMD2I/R9 and potentially the first for any limb-girdle muscular dystrophy, giving BridgeBio a pioneering position in this niche neuromuscular space. The company also plans additional studies in younger LGMD2I/R9 patients and in LGMD2M/2U, signaling an intent to broaden the addressable population over time.

8-K Event Classification

2 items: 8.01, 9.01

2 items

Item 8.01 Other Events Other

Voluntary disclosure of events the company deems important to shareholders but not covered by other items.

Item 9.01 Financial Statements and Exhibits Exhibits

Financial statements, pro forma financial information, and exhibit attachments filed with this report.

Key Figures

FORTIFY interim duration: 12-month interim analysis Loss of ambulation rate: 25% Assisted ventilation rate: 10% +3 more

6 metrics

FORTIFY interim duration 12-month interim analysis Phase 3 FORTIFY trial of BBP-418

Loss of ambulation rate 25% Adults with homozygous L276I LGMD2I/R9 genotype losing independent ambulation

Assisted ventilation rate 10% Adults with homozygous L276I LGMD2I/R9 genotype requiring assisted ventilation

Cardiomyopathy incidence (L276I) 30% Adults with homozygous L276I LGMD2I/R9 genotype developing cardiomyopathy

Annual LVEF decline 0.4% Yearly loss of left ventricular ejection fraction in cardiomyopathy

Cardiac involvement (other FKRP genotypes) 60% Frequency of cardiac involvement in earlier-onset FKRP genotypes

Key Terms

New Drug Application, Orphan Drug Designation, Fast Track designation, Rare Pediatric Disease Designation, +2 more

6 terms

New Drug Application regulatory

"announced the submission of its New Drug Application (NDA) to the FDA"

A new drug application is a formal request submitted to government regulators seeking approval to market a new medicine. It is like a detailed proposal that shows the drug has been tested for safety and effectiveness. For investors, receiving approval signals that the drug may soon become available for sale, potentially leading to revenue growth and impacting the company's value.

Orphan Drug Designation regulatory

"BBP-418 has previously received Orphan Drug, Fast Track, and Rare Pediatric"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

Fast Track designation regulatory

"With Fast Track designation and the potential for BBP-418 to address unmet"

A "fast track designation" is a process that speeds up the review and approval of a product or project, allowing it to reach the market or be completed more quickly than usual. For investors, it can signal that a product may become available sooner, potentially leading to earlier revenue or benefits, and indicating a priority status that might influence company performance and market opportunities.

Rare Pediatric Disease Designation regulatory

"Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation"

A rare pediatric disease designation is an official regulatory status given to a drug or therapy that targets a serious or life‑threatening condition primarily affecting children and is uncommon in the population. It matters to investors because the status often brings financial and development perks — such as tax credits, reduced fees, faster review and periods of market protection — which can lower costs, speed approval and improve the commercial outlook; think of it as a VIP pass that makes bringing a scarce, child‑focused treatment to market easier and potentially more profitable.

Priority Review Voucher regulatory

"if BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher"

A priority review voucher is a transferable regulatory incentive that lets a company move a future drug or device application to the front of the review line, shortening the review period by several months. For investors it matters because the voucher can speed up market access for a high-value product or be sold to other companies for significant cash, acting like a tradable fast-pass that can accelerate revenue or create immediate financial upside.

limb-girdle muscular dystrophy type 2I/R9 medical

"for the treatment of individuals living with limb-girdle muscular dystrophy type 2I/R9"

A genetic form of muscular dystrophy that mainly weakens the muscles around the hips and shoulders and can also affect the heart and breathing; it progresses over years and varies in severity from person to person. For investors, it matters because the clear genetic cause and measurable disease course make it a defined target for drug development, clinical trials, and potential therapies—similar to fixing a specific faulty part in a machine, which helps estimate market size, regulatory hurdles, and commercial opportunity.

Understanding 8-K Material Events →

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): March 30, 2026

BridgeBio Pharma, Inc.

(Exact name of Registrant as Specified in Its Charter)

Delaware	001-38959	84-1850815
(State or Other Jurisdiction of Incorporation)	(Commission File Number)	(IRS Employer Identification No.)

3160 Porter Dr., Suite 250
Palo Alto, CA		94304
(Address of Principal Executive Offices)		(Zip Code)

Registrant’s Telephone Number, Including Area Code: (650) 391-9740

Not Applicable

(Former Name or Former Address, if Changed Since Last Report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, par value $0.001 per share	BBIO	The Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 8.01. Other Events

On March 30, 2026, BridgeBio Pharma, Inc. issued a press release titled “BridgeBio Submits NDA to FDA for BBP-418 for Individuals Living with LGMD2I/R9.” A copy of the press release is attached as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits.

Exhibit	Description
99.1	Press Release titled “BridgeBio Submits NDA to FDA for BBP-418 for Individuals Living with LGMD2I/R9”
104	Cover Page Interactive Data File (embedded within the Inline XBRL document)

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

			BridgeBio Pharma, Inc.
Date:	March 30, 2026	By:	/s/ Thomas Trimarchi
			Thomas Trimarchi, Ph.D.
			President and Chief Financial Officer

Exhibit 99.1

BridgeBio Submits NDA to FDA for BBP-418 for Individuals Living with LGMD2I/R9

- Comprehensive submission includes interim Phase 3 FORTIFY data demonstrating BBP-418’s rapid, consistent treatment effect and favorable safety profile, with statistically significant and clinically meaningful improvements in ambulation and pulmonary function

-Based on ongoing discussions with the FDA, BridgeBio anticipates approval with a U.S. launch anticipated in late 2026/early 2027

-If approved, BBP-418 has the potential to become the first and only therapy for individuals living with LGMD2I/R9 and may represent the first approved treatment for any form of LGMD

PALO ALTO, Calif., March 30, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a biopharmaceutical company focused on developing medicines for genetic conditions, today announced the submission of its New Drug Application (NDA) to the FDA for oral BBP-418 for the treatment of individuals living with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

“This NDA submission brings us one step closer to delivering the first approved therapy to individuals and families affected by LGMD2I/R9, a severe, progressive neuromuscular disease,” said Christine Siu, Chief Executive Officer of BridgeBio Neuromuscular. “This achievement not only reflects the strength of the data, but also our dedicated focus on addressing the urgency of a community that has long been waiting for meaningful treatment options. We are committed to working closely with the FDA to make this potential disease-modifying therapy available as quickly as possible.”

FORTIFY, the Phase 3 clinical trial of BBP-418, successfully met all pre-specified primary and secondary endpoints of its 12-month interim analysis. The topline results can be found here. Additional positive results demonstrating the rapid and consistent treatment effect and favorable safety profile of BBP-418 were presented earlier this month at the 2026 MDA Clinical and Scientific Conference in a late-breaking oral presentation, which can be found here.

BridgeBio anticipates a U.S. launch following FDA approval in late 2026/early 2027. If successful, BBP-418 would be the first and only approved therapy for individuals living with LGMD2I/R9, potentially representing the first approval of a therapy for any form of LGMD. The Company is also engaging regulatory agencies to identify an expedited path to approval for BBP-418 in Europe.

BBP-418 has previously received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency (EMA). With Fast Track designation and the potential for BBP-418 to address unmet need in LGMD2I/R9, the NDA for BBP-418 may be eligible for Priority Review. Consistent with Rare Pediatric Designation from the FDA, if BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher. The Company intends to initiate clinical studies of BBP-418 in LGMD2I/R9 for individuals less than 12 years of age and in LGMD2M/2U in the near future.

About Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) LGMD2I/R9 is a monogenic autosomal recessive disease caused by partial loss of function mutations in the fukutin-related protein (FKRP) gene, and FKRP mutations impair glycosylation of alpha-dystroglycan (αDG), a protein associated with stabilizing muscle cells. Clinical manifestations typically present as a skeletal myopathy affecting the lower and then upper limbs, which is commonly later accompanied by pulmonary muscle and cardiac muscle involvement. Individuals who harbor a homozygous L276I genotype typically develop disease manifestations during late childhood with progression to loss of independent ambulation (25%), assisted ventilation (10%), and cardiomyopathy (30%) in adulthood. Cardiomyopathy is progressive, with an annual loss of 0.4% of left ventricular ejection fraction (LVEF). Individuals with other FKRP genotypes typically have an earlier childhood onset with a more severe clinical course, rapid loss of mobility by 20 years of age, more frequent cardiac involvement (60%), and eventual pulmonary decline by 30 years of age in nearly all cases.

About BridgeBio BridgeBio exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations. Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market. For more information, visit bridgebio.com and follow us on LinkedIn, X, Facebook, Instagram, YouTube, and TikTok.

BridgeBio Forward-Looking Statements

This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements include express and implied statements relating to the Company’s expectations regarding the regulatory review process, potential approval, and timing of a potential launch for BBP-418 in LGMD2I/R9; the Company’s expectations regarding the initiation and timing of clinical trials of BBP-418 in LGMD2I/R9 for individuals less than 12 years of age and in LGMD2M/2U; the potential for BBP-418 to become the first and only approved therapy for LGMD2I/R9 and potentially the first approved therapy for any form of LGMD; the anticipated regulatory pathway for BBP-418; and the potential eligibility of BBP-418 for Priority Review and under the Rare Pediatric Disease Priority Review Voucher program, and the related FDA review timeline. Such statements reflect the Company’s current views about the Company’s plans, intentions, expectations and strategies, which are based on the information currently available to it and on assumptions the Company has made. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, the Company can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from the Company’s clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for the Company’s product candidates, the FDA or such other regulatory agencies not agreeing with the Company’s regulatory approval strategies, components of the Company’s filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, the Company operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of the Company’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact: Bubba Murarka, Executive Vice President contact@bridgebio.com (650)-789-8220

BridgeBio Investor Contact: Chinmay Shukla, Senior Vice President, Strategic Finance ir@bridgebio.com

FAQ

What did BridgeBio Pharma (BBIO) announce about BBP-418?

BridgeBio Pharma submitted a New Drug Application to the FDA for oral BBP-418 to treat limb-girdle muscular dystrophy type 2I/R9. The application is backed by Phase 3 FORTIFY interim data showing significant functional benefits and a favorable safety profile for individuals living with this rare neuromuscular disease.

What is significant about BBP-418 for LGMD2I/R9 according to BBIO?

BBP-418 could become the first and only approved therapy for individuals with LGMD2I/R9. BridgeBio also notes it may represent the first approved treatment for any form of limb-girdle muscular dystrophy, addressing a severe condition with no existing disease-modifying options.

What did the Phase 3 FORTIFY trial show for BridgeBio’s BBP-418?

The Phase 3 FORTIFY trial’s 12‑month interim analysis met all pre-specified primary and secondary endpoints. Data demonstrated rapid, consistent treatment effects, with statistically significant and clinically meaningful improvements in ambulation and pulmonary function, alongside a favorable safety profile in individuals with LGMD2I/R9.

When does BridgeBio (BBIO) anticipate BBP-418 could launch in the U.S.?

BridgeBio anticipates a potential U.S. launch of BBP-418 in late 2026 or early 2027, following FDA approval. This timing reflects ongoing regulatory review expectations and would bring the first targeted therapy to people living with LGMD2I/R9 if approval is granted.

What regulatory designations has BBP-418 received in the U.S. and Europe?

In the U.S., BBP-418 has Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA. In Europe, it holds Orphan Drug Designation from the EMA. These statuses can support expedited development, review, and potential Priority Review and voucher eligibility if the drug is approved.

Is BridgeBio planning further studies of BBP-418 beyond the current NDA population?

BridgeBio intends to initiate clinical studies of BBP-418 in LGMD2I/R9 for individuals under 12 years of age and in LGMD2M/2U. These planned programs aim to extend potential benefit to younger patients and additional limb-girdle muscular dystrophy subtypes if safety and efficacy are confirmed.

Filing Exhibits & Attachments

4 documents

Press Releases

• EX-99.1 EXHIBIT 99.1 17.1 KB

Other Documents

• EX-101 XBRL TAXONOMY EXTENSION SCHEMA 3.8 KB
• EX-101 XBRL TAXONOMY EXTENSION LABEL LINKBASE 21.5 KB
• EX-101 XBRL TAXONOMY EXTENSION PRESENTATION LINKBASE 15.7 KB