PRINCETON, New Jersey, U.S. — OCTOBER 22, 2025 — Ascendis Pharma Endocrinology, Inc. today announced that SKYTROFA® (lonapegsomatropin-tcgd), its once-weekly growth hormone replacement therapy, is now available in broader dosing ranges in the United States (U.S.) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD). The broader ranges are designed to support physicians titrating dosing for adults with GHD who are beginning growth hormone treatment for the first time. SKYTROFA was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with GHD, a rare and serious condition, in July 2025 and for the treatment of pediatric GHD in August 2021.
“Growth hormone deficiency in adults can be quite debilitating and stigmatizing and is often misdiagnosed when its physical and psychological symptoms are attributed to other more common conditions,” said Lisa Abbott, M.D., board certified endocrinologist at Northern Nevada Endocrinology in Reno, Nevada. “With these broader SKYTROFA dosing ranges, healthcare providers can offer this clinically proven, safe and convenient once-weekly alternative to daily injections to adults just initiating therapy or for adolescents transitioning into adult care.”
In the U.S., GHD affects an estimated two to three of every 10,000 adults. With this condition, a person’s pituitary gland does not produce enough growth hormone. Clinical symptoms can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression. GHD can develop for the first time in adulthood or can start in childhood and continue into adulthood. It often results from surgery or radiation to or near the pituitary gland or from severe head trauma.
“Development of SKYTROFA in dosing increments that meet the needs of more patients underscores our commitment to making a meaningful difference in the lives of patients impacted by this rare, serious, and often misunderstood condition,” said Jay Donovan Wu, Executive Vice President and President, U.S. Market at Ascendis Pharma. “SKYTROFA was the first once-weekly long-acting growth hormone treatment introduced in the U.S. for pediatric GHD and we are proud to now offer this trusted therapy in dosing increments optimized for adults with GHD.”
The FDA’s approval of SKYTROFA for adults with GHD was based on results from foresiGHt, Ascendis Pharma’s Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) clinical trial that compared the efficacy and safety of weekly TransCon hGH (lonapegsomatropin-tcgd, now approved as SKYTROFA®) with weekly placebo and daily somatropin in adults with GHD.
To support the patient treatment journey, Ascendis has established a dedicated SKYTROFA team within the U.S. Ascendis Signature Access Program®, which is staffed by specially trained nurses and offers a full suite of programs designed to help patients, caregivers, and physicians navigate each step of the treatment process. These include clinical education, assistance with prior authorization and appeals, training on proper injection procedures, and co-pay and other related assistance for eligible patients.
About SKYTROFA Administration
The SKYTROFA® Auto-Injector and cartridges are uniquely designed to deliver a full week’s dose and minimize the potential for wasted medication with no split dosing or dose dialing and no exposed needles during injections. After first removal from a refrigerator, the medicine can be stored at room temperature for up to six months. With a weekly injection, patients switching from injections every day can experience up to 86 percent fewer injection days per year.
About TransCon Technology
At the core of Ascendis Pharma’s innovative approach is TransCon, our flexible technology platform that combines the benefits of conventional prodrug and sustained release technologies to solve the fundamental limitations seen in other approaches to extending the duration of a drug’s action in the body. Each TransCon prodrug molecule has up to three components: a parent drug with known biology, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected, physiologic conditions in the body initiate the release of the active, unmodified parent drug in a predictable manner at a predetermined rate.
About Adult Growth Hormone Deficiency
Growth hormone plays an essential role in the health of children and adults, promoting normal growth in children and maintenance of normal body composition and cardiometabolic health throughout adulthood. In adults, growth hormone boosts protein production, promotes fat utilization, enhances muscle mass, and helps regulate blood sugar levels. Adult GHD is a condition in which an individual’s body does not produce enough growth hormone. Symptoms and morbidity can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression.
About Ascendis Pharma Endocrinology, Inc.
Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. This includes the U.S. Market operations based in Princeton, New Jersey. Please visit ascendispharma.us to learn more about our commercial operations in the United States.
The following information is intended for the U.S. Audience Only:
IMPORTANT SAFETY INFORMATION & USES
SKYTROFA is a prescription medicine used for:
- the replacement of growth hormone in children 1 year old or older who weigh at least 26 pounds (11.5 kilograms) with growth hormone deficiency (GHD)
- the replacement of growth hormone in adults with growth hormone deficiency (GHD)
Do not take SKYTROFA if:
- you have a critical illness caused by certain types of heart or stomach surgery, trauma or breathing problems
- you are allergic to somatropin or any of the ingredients in SKYTROFA
- you are a child with closed bone growth plates
- you have cancer or other tumors
- you have certain types of eye problems caused by diabetes
- you are a child with Prader-Willi syndrome who is severely obese or has breathing problems including sleep apnea (briefly stopping breathing during sleep)
Tell your healthcare provider if you are pregnant or plan to become pregnant, about all of your medical conditions, and about all the medicines you take. SKYTROFA may affect how other medicines work, and other medicines may affect how SKYTROFA works.
What are the possible side effects of SKYTROFA?
SKYTROFA may cause serious side effects, including:
- serious allergic reactions. Get medical help right away if you have swelling of the face, mouth, tongue, or throat or trouble breathing
- high risk of death in people who have critical illnesses caused by heart or stomach surgery, trauma, or serious breathing problems
- increased risk of growth of an existing tumor or cancer, or increased risk of a previous tumor or cancer returning in people who were treated with radiation to the brain or head as children and who developed low growth hormone problems. Your healthcare provider will need to monitor for the growth or return of a tumor or cancer. Contact your healthcare provider if you start to have sudden changes in behavior, headaches, vision problems, or changes in moles, birthmarks, or skin color
- new or worsening high blood sugar or diabetes. Your blood sugar may need to be monitored during treatment with SKYTROFA
- increase in pressure in the skull. Contact your healthcare provider if headaches, eye problems, nausea, or vomiting occur
- too much fluid in the body (fluid retention). Contact your healthcare provider if you notice swelling in the hands and feet, pain in the joints or muscles, or nerve problems that cause pain, burning or tingling in the hands, arms, legs, and feet
- decrease in a hormone called cortisol. Your healthcare provider will do blood tests to check your cortisol levels
- decrease in thyroid hormone levels. Decreased thyroid hormone levels may affect how well SKYTROFA works. Your healthcare provider will do blood tests to check your thyroid hormone levels
- hip and knee pain or a limp in children (slipped capital femoral epiphysis). This may lead to a serious condition where bone tissue dies due to a lack of blood supply (osteonecrosis). Get medical help right away for children who develop a limp or have hip or knee pain
- worsening of curvature of the spine in children (scoliosis)
- severe and constant abdominal pain. This could be a sign of pancreatitis. Tell your healthcare provider about any new abdominal pain
- loss of fat and tissue weakness in the area of skin you or your child inject. Talk to your healthcare provider about rotating the areas where SKYTROFA is injected
- high risk of sudden death in children with Prader-Willi syndrome who are severely obese or have breathing problems, including sleep apnea
- increase in phosphate, alkaline phosphatase, and parathyroid hormone levels in your blood. Your healthcare provider will do blood tests to check this
The most common side effects of SKYTROFA in children include: viral infection, fever, cough, nausea and vomiting, bleeding, diarrhea, stomach area pain, joint pain and arthritis
The most common side effects of SKYTROFA in adults include: swelling due to fluid build-up and low thyroid hormone
These are not all of the possible side effects of SKYTROFA. Call your doctor for medical advice about side effects. You are encouraged to report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch. You may also report side effects to Ascendis Pharma at 1-844-442-7236.
Please click here for SKYTROFA full Prescribing Information.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and SKYTROFA® are trademarks owned by the Ascendis Pharma group.
© October 2025 Ascendis Pharma Endocrinology, Inc.
U.S. Market Media Contact:
Ascendis Pharma Endocrinology, Inc.
Dana Lynch, Senior Director, U.S. Communications and Public Relations