This week's biotech landscape witnessed key FDA approvals, rejections, NDA resubmissions, merger terminations, trial discontinuations, and clinical trial data readouts across therapeutic areas such as Turner Syndrome, Late-Line Colorectal Cancer, Obesity, and Type 1 Diabetes. Let us unpack the key developments and milestones in the biotech space this week. FDA and EU Approvals & Rejections GSK's Lynavoy Wins FDA Approval for Cholestatic Pruritus in PBC GSK plc (GSK), secured for Lynavoy, the first U.S. tre ...

- Annualized height velocity of 9.05 cm/year (LS mean) for TransCon hGH-treated children was similar at Week 52 to daily somatropin-treated children - TransCon hGH showed a safety and tolerability profile comparable to daily somatropin with no occurrences of slipped capital femoral epiphysis (SCFE) COPENHAGEN, Denmark, March 17, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced positive Week 52 topline results from New InsiGHTS, its Phase 2 randomized, open-label, active-controll ...

COPENHAGEN, Denmark, March 16, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced new data from its pivotal ApproaCH Trial showing that children with achondroplasia treated with once-weekly TransCon CNP (navepegritide) maintained consistent improvements in growth through Week 104, with further improvement in body proportionality during the second year of treatment. These and other results were shared in an oral presentation by Dr. Ravi Savarirayan, M.B.B.S., M.D., Murdoch Children’s ...

Core Insights - The FDA has granted multiple approvals for new treatments across various therapeutic areas, including oncology and rare genetic disorders, in late February and early March 2026 Group 1: Boehringer Ingelheim Pharmaceuticals Inc. - The FDA granted accelerated approval to Hernexeos as a first-line treatment for adult patients with advanced non-small cell lung cancer (NSCLC) with HER2 mutations on February 26, 2026 [2] - Hernexeos is the first targeted therapy for HER2-mutant advanced NSCLC in the first-line setting, with HER2 mutations occurring in approximately 2-4% of NSCLC cases [3] Group 2: Ascendis Pharma A/S - Ascendis received accelerated approval for Yuviwel, a once-weekly treatment for children aged 2 years and older with achondroplasia on February 27, 2026 [4] - Yuviwel is the first and only approved therapy providing continuous systemic exposure to C-type natriuretic peptide (CNP) over a weekly dosing interval [6] Group 3: BioMarin Pharmaceutical Inc. - BioMarin expanded the indication of Palynziq to include pediatric patients aged 12 years and older with phenylketonuria (PKU) on February 27, 2026 [7] - Palynziq is the only enzyme substitution therapy approved to reduce blood phenylalanine concentrations in PKU patients, generating $433 million in revenue in 2025, up from $355 million in 2024 [9] Group 4: Novo Nordisk A/S - Novo Nordisk received FDA approval for three new indications for Sogroya, a long-acting growth hormone, on February 27, 2026 [10] - Sogroya is now approved for children aged 2.5 years and older with Idiopathic Short Stature, short stature born Small for Gestational Age, and growth failure associated with Noonan Syndrome [11] - The once-weekly administration of Sogroya aims to improve adherence compared to daily growth hormone injections [12] Group 5: Johnson & Johnson - The FDA approved Tecvayli plus Darzalex Faspro for adults with relapsed or refractory multiple myeloma on March 5, 2026 [13] - Tecvayli received accelerated approval in October 2022 as a subcutaneous treatment for patients with RRMM who have undergone multiple prior therapies [14] - Darzalex Faspro is already approved for multiple indications in multiple myeloma, enhancing treatment options for patients [15]

Jefferies sets a price target of $262 for NASDAQ:ASND, indicating a potential upside of approximately 10.39%.The company's participation in the TD Cowen 46th Annual Health Care Conference highlights its growth prospects.ASND's current trading price is $237.35, with a year's trading range between $124.06 and $248.60, showcasing volatility.Ascendis Pharma A/S (NASDAQ:ASND) is a biopharmaceutical company focused on developing therapies for rare diseases. The company is known for its innovative TransCon technol ...

Question-and-Answer SessionYaron WerberTD Cowen, Research Division So the conference call, I think, literally ended about 2 hours ago. The label looks very clean. As you guys showed the slide and the AEs are minimal. The nice feature of the drug is that it's weekly. It can actually be kept at room temperature for up to 6 months and then can actually be put back in the fridge. That's a slight differentiator also from VOXZOGO. The label is 2 years old. You'll show us the long-term extension later on. And I th ...

Core Viewpoint - Ascendis Pharma has received FDA approval for a new treatment, positioning the company for potential growth as it nears a buy point in the stock market [1] Company Overview - Ascendis Pharma is a Denmark-based biotech company with three FDA-approved products developed using its TransCon technology platform, which aims to enhance treatment efficacy, tolerability, and convenience [1] - The company recently received FDA approval for Yuviwel, a weekly treatment for children with achondroplasia, a rare genetic condition affecting bone growth [1] - Other products include Skytrofa for pediatric growth hormone deficiency and Yorvipath for hypoparathyroidism, with a treatment for Turner syndrome currently in Phase 2 trials [1] Market Performance - Ascendis Pharma's stock reached an all-time high of $248.60 but retreated by 1.2% as the market fell [1] - The stock is currently trading around 2% below a buy point, with a relative strength line reaching a 52-week high [1] - As of December, 509 mutual funds owned shares of Ascendis, an increase from 493 in September, indicating growing institutional interest [1] Financial Performance - In the fourth quarter, Ascendis reported a loss of $0.65 per share on revenue of $290.7 million [1] - Analysts project the company will turn a profit of $0.40 per share in the first quarter, with earnings expected to increase to $0.88, $1.45, and $2.27 per share over the next three quarters [1] - Full-year 2027 profit forecasts from Wall Street estimate earnings of $12.34 per share, with a three-year sales growth rate of 122% [1]

Core Insights - The Growth Stock Forum focuses on identifying attractive growth stocks, particularly in the biotech sector, emphasizing risk/reward situations [1] - The biotech Top Picks of the forum achieved a remarkable increase of 137.6% last year, with a compound annual growth rate (CAGR) of 37.2% since 2017 [1] - The core portfolio, LTGP, has also demonstrated strong performance with a mid-20s CAGR as of February 20, 2026 [1]

Core Viewpoint - Ascendis Pharma A/S has received an upgrade from Evercore ISI to "Outperform" with a price target of $324, driven by the FDA's approval of its innovative therapy Yuviwel for achondroplasia in children [1][6]. Group 1: Company Overview - Ascendis Pharma A/S is a biopharmaceutical company focused on developing therapies for rare diseases [1]. - The company's current stock price is approximately $233, with a market capitalization of around $14.3 billion [4]. Group 2: Product Development - The FDA approved Yuviwel, a once-weekly treatment for children with achondroplasia, based on data from three randomized clinical trials [2]. - Yuviwel is the first therapy designed to enhance linear growth in children aged two years and older, marking a significant advancement in treatment options for this rare genetic disorder [2][3]. Group 3: Regulatory Milestones - The approval of Yuviwel under the FDA's Accelerated Approval Program is a major milestone for Ascendis Pharma, emphasizing the therapy's importance [3]. - The company has also received a Rare Pediatric Disease Priority Review Voucher, further highlighting the significance of Yuviwel [3]. Group 4: Market Performance - The stock of ASND has shown fluctuations, with a trading volume of 602,078 shares, indicating strong investor interest [5]. - The stock has a 52-week high of $242 and a low of $124.06, reflecting its volatility in the market [5].

Core Viewpoint - The conference call is focused on the FDA approval of YUVIWEL, highlighting its significance for Ascendis Pharma and its stakeholders [1]. Group 1: Company Overview - Chad Fugure serves as the Vice President of Investor Relations for Ascendis Pharma, leading the conference call [2]. - Key executives present include Jan Mikkelsen (President and CEO), Scott Smith (EVP and CFO), Jay Wu (EVP, President, U.S. Market), Aimee Shu (Chief Medical Officer), and Sherrie Glass (Chief Business Officer) [2]. Group 2: Conference Call Details - The call is being recorded, indicating a formal communication process for stakeholders [1]. - Forward-looking statements will be included in the discussion, suggesting a focus on future expectations and projections for the company [3].