Infigratinib Phosphate

- This voluntary agreement expands access for Medicaid patients to BridgeBio’s currently marketed medicine and lowers drug costs for Americans without jeopardizing innovation and sustainability in rare diseases - BridgeBio will continue to offer Attruby ® via Medicare Part D without any future pricing mandates - The agreement has no impact on ForgingBridges ® , a copay assistance program that helps reduce out-of-pocket costs to as little as $0 per month for qualifying patients PALO ALTO, Calif., Aug. 31, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, today announced that it has entered into a voluntary agreement with the U.S. government to expand access to its medicines and lower costs for American patients. Neil Kumar, Ph.D., Co-Founder and CEO of BridgeBio, joined President Donald J. Trump and members of his Administration at the White House to discuss the new agreement, which improves access to treatments for rare genetic diseases without jeopardizing innovation or sustained investment. Millions of people worldwide live with rare genetic conditions that have no approved treatment options because developing medicines for rare diseases has never been commercially straightforward. Today’s agreement with the Administration is intended to ensure that BridgeBio will be able to continue bringing medicines to people living with rare genetic diseases. As part of the agreement, BridgeBio will expand state Medicaid access to its currently marketed medicine via the GENEROUS Model. This builds on the Company’s existing patient access work, including ForgingBridges, BridgeBio’s patient support program, which provides reimbursement navigation and financial assistance to qualifying patients, potentially minimizing out-of-pocket costs to as little as $0 per month. BridgeBio does not expect to be subject to future pricing mandates. The specific terms of the agreement remain confidential. “As an American biotech, it’s a privilege to be working alongside the Administration to ensure the broadest possible access for Americans to the medicines that we make. Thirty million Americans suffer from rare genetic disorders, and our intent is to reliably innovate new medicines and bring them to as many communities as possible,” said Dr. Kumar. “Within the field of ATTR-CM, we’ve already launched the lowest-priced product with the best data at 30 months, and we continue to look forward to working with anyone who wants to help improve access to treatment for the patients who need it.” BridgeBio’s model was built to make drug development and innovation economically viable for genetic conditions that affect small patient populations. The Company’s approved medicine, Attruby, is available to people with transthyretin amyloid cardiomyopathy, and the Company has three additional medicines under FDA review, each for a genetic condition with limited or no approved treatment options: BBP-418 for limb-girdle muscular dystrophy type 2I/R9, or LGMD2I/R9 (PDUFA date with Priority Review: November 27, 2026); encaleret for autosomal dominant hypocalcemia type 1, or ADH1 (PDUFA date with Priority Review: May 7, 2027); and infigratinib for achondroplasia. 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 . About Attruby ® (acoramidis) INDICATION Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. IMPORTANT SAFETY INFORMATION Adverse Reactions Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). 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 statements regarding the anticipated implementation, scope and effects of BridgeBio’s agreement with the U.S. government, including BridgeBio’s plans to expand Medicaid access to Attruby through the GENEROUS Model; BridgeBio’s expectation that it will not be subject to future pricing mandates; and the anticipated impact of the agreement on patient access, affordability and BridgeBio’s ability to continue developing and providing medicines for rare genetic diseases. 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, the risk that the agreement may be implemented, interpreted or applied differently than BridgeBio currently expects; that federal or state laws, regulations, policies, reimbursement frameworks or government pricing programs may change or be implemented in a manner that adversely affects BridgeBio or its products; that BridgeBio may become subject to additional pricing mandates, requirements or restrictions notwithstanding its current expectations that the agreement may not result in the anticipated improvements in access, affordability or other expected benefits; that the agreement or future changes in government pricing or reimbursement policies may adversely affect BridgeBio’s business, results of operations or ability to continue investing in the development and commercialization of medicines for rare genetic diseases; the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Middle East, 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 Quarterly Report on Form 10-Q and 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: Kaitlyn Reilly, Director, Communications contact@bridgebio.com (650) 789-8220 BridgeBio Investor Contact: Kristen Kelleher, Director, Investor Relations ir@bridgebio.com

iStock, drogatnev BioMarin’s discontinued drug showed biomarker benefits in a Phase 3 study for ENPP1 deficiency but these failed to translate to clinical improvements. BioMarin will no longer invest in an investigational enzyme replacement therapy following a disappointing late-stage showing earlier this year. The asset, dubbed BMN 401, was being studied in the Phase 3 ENERGY trial for ENPP1 deficiency , a rare metabolic disease that involves a range of symptoms, including early-onset osteoporosis, the calcification of tendons and a higher risk of cardiovascular death. The candidate came to BioMarin in May 2025 as the centerpiece of its $270 million takeover of Inozyme Pharma. A year later, however, the asset turned out to be a disappointment, meeting only one of two key endpoints in ENERGY. While BMN 401 significantly lowered levels of a key disease marker, this effect failed to translate into clinical benefit for patients. ezstandalone.cmd.push(function () { ezstandalone.showAds(129); }); The ENERGY failure has prompted BioMarin to discontinue the development of BMN 401 across all indications, according to the company’s second quarter earnings report released on Thursday. In addition to ENPP1 deficiency, Inozyme had previously been testing the molecule for ABCC6 deficiency and calcific uremic arteriopathy, both rare diseases. BioMarin no longer lists any active program in these indications. Rare diseases BioMarin suffers another blow to rare disease portfolio in Phase 3 flop BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth. May 19, 2026 · 2 min read · Tristan Manalac Read more Other than the discontinuation, BioMarin reported positive earnings results this quarter. Revenues climbed 20% year-on-year to hit $990 million, the biopharma reported. BioMarin’s product portfolio was led by the achondroplasia medicine Voxzogo, which brought in $253 million. Of note, Voxzogo sales continued to grow—14% versus the same period in 2025—despite the entry of a key competitor: Ascendis Pharma’s Yuviwel, which won FDA approval in achondroplasia, the most common cause of dwarfism, this past March. Ascendis is scheduled to present its second quarter earnings on Aug. 13. “Voxzogo continues to offset any impact with growth in the <2 [years] population and in the ex-US population, which comprises the majority of revenues,” Stifel analysts wrote in a note on Friday. “The next couple quarters will be key here in determining the competitive impact to BioMarin.” In October last year, in anticipation of Yuviwel’s approval, BioMarin withdrew a previously announced goal of hitting $4 billion in revenue by 2027—a decision driven largely by the impact of potential challengers to Voxzogo. On Thursday, however, BioMarin’s confidence in Voxzogo appeared to grow, raising full year sales guidance from a range of $975 million to $1.02 billion to a minimum of $1 billion with the potential to reach $1.05 billion. Aside from Ascendis, BridgeBio Pharma is also advancing on the achondroplasia market with its investigational pill dubbed infigratinib . Data released in late June showed that aside from boosting growth in children, infigratinib also improved body proportionality , a finding that could help the drug stand out in the achondroplasia arena. A regulatory filing for infigratinib is planned for the third quarter, with a potential launch in early to mid-2027. Earnings BioMarin Abandons Gene Therapy Roctavian, Cuts Revenue Guidance as Voxzogo Faces Competitors BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending competition for achondroplasia therapy Voxzogo and the divestment of the gene therapy Roctavian. October 28, 2025 · 4 min read · Tristan Manalac Read more