Ascendis Pharma A/S
SKYTROFA® FDA Approval
August 25, 2021
For investor communication only. Not for use in product promotion. | ||
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Cautionary Note On Forward-Looking Statements
This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, such as statements regarding our future results of operations and financial position, including our business strategy, expectations regarding the potential benefits of Skytrofa, expectations regarding the projected timing of availability of Skytrofa, expectations regarding a patient support services, availability of funding, clinical trial results, product approvals and regulatory pathways, collaborations, licensing or other arrangements, the potential market size and size of the potential patient populations for Skytrofa and our other product candidates, timing and likelihood of success, plans and objectives of management for future operations, our ability to integrate data informatics to create innovative patient care solutions and enhance patient care, the scope, progress, results and costs of developing our product candidates or any other future product candidates, and future results of current and anticipated products are forward-looking statements. These forward-looking statements are based on our current expectations and beliefs, as well as assumptions concerning future events. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the results discussed in the forward-looking statements. These risks, uncertainties and other factors are more fully described in our reports filed with or submitted to the Securities and Exchange Commission, including, without limitation, our most recent Annual Report on Form 20-F filed with the SEC on April 3, 2020 particularly in the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". In light of the significant uncertainties in our forward-looking statements, you should not place undue reliance on these statements or regard these statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified timeframe, or at all.
Any forward-looking statement made by us in this presentation speaks only as of the date of this presentation and represents our estimates and assumptions only as of the date of this presentation. Except as required by law, we assume no obligation to update these statements publicly, whether as a result of new information, future events, changed circumstances or otherwise after the date of this presentation.
Skytrofa has been approved by the U.S. Food and Drug Administration for the treatment of pediatric growth hormone deficiency. Skytrofa is and has been under clinical investigation and has not yet been approved for marketing by the European Medicines Agency or other foreign regulatory authorities.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are
2 trademarks owned by the Ascendis Pharma Group. © August 2021 Ascendis Pharma A/S.
For investor communication only. Not for use in product promotion. Do not distribute.
SKYTROFA® Now FDA Approved
• First FDA-approvedonce-weekly product for pediatric growth hormone deficiency (GHD)
• First FDA-approved product utilizing TransCon™ technology
For investor communication only. Not for use in product promotion. | ||
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Skytrofa: Select Highlights of U.S. Prescribing Information
INDICATIONS AND USAGE | Skytrofa is a human growth hormone indicated for the treatment of pediatric patients 1 year and older | ||||
who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth | |||||
hormone (GH) | |||||
DOSAGE AND ADMINISTRATION | Skytrofa should be administered subcutaneously into the abdomen, buttock, or thigh with regular | ||||
rotation of the injection sites. | |||||
The recommended dose is 0.24 mg/kg body weight once-weekly. | |||||
Safety | Skytrofa is contraindicated in patients with: | ||||
• | Acute critical illness after open heart surgery, abdominal surgery or multiple accidental | ||||
trauma, or those with acute respiratory failure due to the risk of increased mortality with use of | |||||
pharmacologic doses of somatropin | |||||
• | Hypersensitivity to somatropin or any of the excipients in Skytrofa | ||||
• | Children with closed epiphyses | ||||
• | Active malignancy | ||||
• | Active proliferative or severe non-proliferativediabetic retinopathy | ||||
• | Prader-Willisyndrome who are severely obese, have a history of upper airway obstruction or | ||||
sleep apnea or have severe respiratory impairment due to the risk of sudden death | |||||
Adverse Reactions: Most common adverse reactions (≥5%) were viral infection (15%), pyrexia (15%), | |||||
cough (11%), nausea and vomiting (11%), hemorrhage (7%), diarrhea (6%), abdominal pain | |||||
(6%), and arthralgia and arthritis (6%). | |||||
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Do not distribute. | |||||
Limited Innovation Since rhGH was Introduced >30 Years Ago
First FDA approval of
daily recombinant human GH (1985)
First FDA approval of a hGH with room-temperature storage (2006)
First pre-filled injection pen in
the US (2004)
First FDA approval of a once-weekly
treatment for pediatric growth hormone deficiency with in-use room temperature storage for 6 months (2021)
1985 | 1990 | 1995 | 2000 | 2005 | 2010 | 2015 | 2020 | 2025 | ||
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Ascendis Pharma A/S published this content on 25 August 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 25 August 2021 20:40:08 UTC.