>> ALL RIGHT WELCOME BACK EVERYBODY. LET'S TALK ALL THINGS PUBLIC HEALTH THIS MORNING INCLUDING THE FIGHT BETWEEN NOVO NORDISK AND HIMS AND HERS OVER GLP ONE DRUGS. JOINING US RIGHT NOW IS CNBC CONTRIBUTOR DOCTOR SCOTT GOTTLIEB.HE IS THE FORMER COMMISSIONER OF THE FDA. AND HE SERVES ON THE BOARDS OF BOTH ILLUMINA PFIZER AND UNITEDHEALTH. HE'S ALSO ON THE CNBC CURES ADVISORY BOARD.AND SCOTT, THANK YOU FOR COMING IN TODAY. THANKS A LOT. BREAK THIS DOWN FOR US HIMS AND HERS.BIG SPLASHY ANNOUNCEMENT LAST WEEK T ...

Leerink analyst Roanna Ruiz was not surprised that Aquestive Therapeutics (AQST) received a Complete Response Letter for Anaphylm given the FDA’s prior deficiency letter. That said, the “silver lining” is that this outcome appears recoverable with a relatively defined path to approval, the firm adds. The good news is that no additional studies beyond these seem to have been requested by the FDA, the CRL does not appear to raise any concerns about comparability data or CMC, and Leerink sees these studies ar ...

Deficiencies limited to packaging and administrationCompany believes it can rapidly resolve deficiencies and expects to resubmit as early as Q3 2026Remains well-capitalized and anticipates ending 2026 with significant cashReiterates plans to submit in Canada and EU by the end of 2026Company to host investor call on February 2, 2026, at 8:00am ET WARREN, N.J., Feb. 02, 2026 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST) ("Aquestive" or the "Company"), a pharmaceutical company advancing medic ...

Unicycive Therapeutics (UNCY) announced on Thursday that the U.S. Food and Drug Administration (FDA) has accepted its resubmitted New Drug Application (NDA) for its kidney disease therapy oxylanthanum carbonate (OLC). In late June, the FDA declined to approve the oral phosphate ...

Core Viewpoint - The FDA has postponed its decision on Disc Medicine Inc.'s experimental treatment, bitopertin, for erythropoietic protoporphyria to February 10, raising questions about the drug's efficacy and potential risks [1][2][3]. Group 1: FDA Review Process - The FDA reviewers have delayed their decision on bitopertin by approximately two weeks, now set for February 10 [1]. - Regulators are questioning the reliability of "pain-free time in the sun" as a secondary endpoint for effectiveness, suggesting that biomarker data may be more appropriate for supporting approval [2]. - FDA staff are evaluating whether bitopertin poses any risk of misuse or addiction, adding complexity to the review process [3]. Group 2: Market Reaction and Analyst Insights - Disc Medicine's stock has seen positive movement, with a reported increase of 2.59% to $72.88 at the time of publication [5]. - Analyst Douglas Tsao from H.C. Wainwright expressed confidence in the likelihood of approval despite the ongoing review process, indicating that any potential delay would be modest [5]. - H.C. Wainwright maintains a Buy rating on Disc Medicine with a price target of $118 [5].

Core Viewpoint - Aquestive Therapeutics Inc. is experiencing a decline in stock price due to identified deficiencies in the New Drug Application for Anaphylm, which is a polymer matrix-based epinephrine prodrug product candidate aimed at treating severe allergic reactions, including anaphylaxis [2][3][6] Group 1: Stock Performance - The stock of Aquestive Therapeutics is trading lower, with a session volume of 5.68 million compared to the average volume of 2.79 million [1] - At the time of publication, shares were down 40.18% at $3.71 [6] Group 2: FDA Review and Deficiencies - The FDA has identified deficiencies in Anaphylm's New Drug Application that prevent discussions on labeling and post-marketing commitments [2] - The specific deficiencies have not been disclosed, but the company is actively working to understand and resolve these concerns [2][4] - The FDA's review of the application remains ongoing, and a Discipline Review Letter will not be issued, although information requests may occur during the review period [4] Group 3: Product Details and Future Plans - Anaphylm is designed to be a sublingual film that is similar in size to a postage stamp and begins to dissolve on contact, requiring no water or swallowing for administration [3] - The company plans to submit for regulatory approval of Anaphylm in Canada, Europe, and the U.K. by 2026 [3] Group 4: Financial Position and Future Outlook - As of the end of 2025, the company had approximately $120 million in cash and cash equivalents, which is believed to be sufficient for completing the Anaphylm approval and launch processes in the U.S. if approved [5] - Delays in communicating specific deficiencies may lead to further delays in the potential approval of Anaphylm [5]

Core Viewpoint - President Trump has threatened to impose a 100% tariff on pharmaceutical companies unless they establish manufacturing plants in the United States, with the tariffs set to take effect on October 1st [1]. Group 1: Impact on Pharmaceutical Companies - The imposition of tariffs will increase costs for pharmaceutical companies, ultimately burdening US consumers and insurance companies [2][3]. - Many early-stage biotech companies rely on outsourced manufacturing to remain cost-competitive during the clinical trial process, which could be jeopardized by tariffs [3]. - Major pharmaceutical companies like Eli Lilly and Novartis are committing to invest over $20 billion in new manufacturing plants in the US, which is seen as positive for the US economy in the long term [4]. Group 2: Short-term Concerns - The immediate concern is the disruption of supply chains and the potential delays in bringing new drugs to market due to the lack of existing manufacturing capacity in the US [5]. - There are mixed signals regarding the FDA's willingness to approve new therapies, which raises concerns among investors about the viability of investing in biotech [8][12]. Group 3: Regulatory Environment - The FDA is reportedly looking to accelerate the drug approval process, which could be favorable for investors and patients [12]. - However, there are concerns about NIH funding and the overall clarity of the regulatory environment, which complicates investment decisions [12][13]. Group 4: Global Trade Considerations - The EU has expressed expectations that the US will respect commitments on pharmaceutical tariff caps, indicating potential trade advantages for EU companies [6][7]. - Countries like Japan and Korea may be shielded from these tariffs due to existing trade agreements [14].

Core Points - Vanda Pharmaceuticals Inc. achieved a significant legal victory against the FDA regarding the approval of HETLIOZ® (tasimelteon) for treating jet lag disorder [1][4] - The U.S. Court of Appeals for the D.C. Circuit ruled that the FDA must engage meaningfully with the evidence presented by drug innovators like Vanda [4][6] - The court's decision mandates that the FDA either approve the supplemental New Drug Application (sNDA) or conduct a hearing on the matter [4][6] Company Summary - Vanda submitted its sNDA in October 2018, but the FDA delayed the resolution, leading to a federal court ruling that the FDA violated the Food, Drug, and Cosmetic Act [2][3] - The court found that Vanda provided substantial evidence of tasimelteon's efficacy in improving sleep disturbances, with trials showing statistically significant improvements [4][5] - Vanda has invested over a decade in clinical studies to develop HETLIOZ®, which has the potential to significantly benefit individuals suffering from jet lag [5][6] Industry Impact - The court's ruling alters the relationship between the FDA and pharmaceutical companies, emphasizing the need for the FDA to fairly evaluate scientific evidence [6][7] - This decision may encourage other drug manufacturers to challenge FDA decisions that they believe are unjust, potentially leading to more innovative therapeutics reaching the market [7]

Core Insights - Vanda Pharmaceuticals reported a significant increase in Fanapt revenue, driven by expanded sales efforts and a direct-to-consumer brand awareness campaign, with expectations for continued growth in upcoming quarters [2][6] - The company anticipates important regulatory and clinical milestones that could enhance its commercial portfolio and advance its clinical pipeline [2] Financial Highlights - In Q2 2025, net product sales from Fanapt® reached $29.3 million, a 27% increase from $23.2 million in Q2 2024 [6][7] - Total net product sales from Fanapt®, HETLIOZ®, and PONVORY® were $52.6 million in Q2 2025, a 4% increase compared to $50.5 million in Q2 2024 [7] - The net loss for Q2 2025 was $27.2 million, compared to a net loss of $4.5 million in Q2 2024 [9] - Cash and cash equivalents stood at $325.6 million as of June 30, 2025, reflecting a decrease of $15.4 million from March 31, 2025 [7] Operational Highlights - Bysanti™ NDA for bipolar I disorder and schizophrenia is under FDA review with a PDUFA target action date of February 21, 2026 [6][8] - Tradipitant NDA for motion sickness is also under review, with a PDUFA target action date of December 30, 2025 [6][14] - Fanapt® experienced a 24% increase in total prescriptions (TRx) in Q2 2025 compared to Q2 2024 [7] Financial Guidance - Vanda expects total revenues for the full year 2025 to be between $210 million and $250 million, with year-end cash projected to be between $280 million and $320 million [11]

Core Viewpoint - A class action has been filed against UroGen Pharma Ltd. for allegedly failing to conduct a proper trial for its lead drug candidate, UGN-102, which is intended to treat low-grade intermediate risk non-muscle invasive bladder cancer [1][2]. Group 1: Allegations and FDA Concerns - The complaint alleges that UroGen submitted a rolling new drug application (NDA) for UGN-102 on August 14, 2024 [2]. - On May 16, 2025, the FDA expressed doubts about the sufficiency of the submitted data for UGN-102's effectiveness, highlighting the lack of a concurrent control arm in the ENVISION clinical study [3]. - The FDA had previously recommended a randomized trial design to UroGen multiple times, which the company did not follow [3]. Group 2: Stock Price Impact - Following the FDA's briefing document on May 16, 2025, UroGen's stock price fell by $2.54, or 25.8%, closing at $7.31 per share [3]. - After the Oncological Drugs Advisory Committee voted against the approval of UGN-102 on May 21, 2025, UroGen's stock price dropped by $3.37, or 44.7%, closing at $4.17 per share [4]. Group 3: Class Action Details - The plaintiff claims that UroGen failed to disclose critical information regarding the study design and its implications for the NDA approval process [5]. - Shareholders interested in participating in the class action can contact Robbins LLP, which is leading the litigation [6].