0LIF Ultragenyx Pharmaceutical Inc

MEDIA ADVISORY: Ultragenyx Announces Mepsevii® (vestronidase alfa) Receives Reimbursement Approval for Treatment of Mucopolysaccharidosis VII in Italy
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Mepsevii® (vestronidase alfa) has been approved for reimbursement by the Agenzia Italiana del Farmaco (Italian Medicines Agency – AIFA) for the treatment of pediatric and adult patients with Mucopolysaccharidosis VII (MPS VII; Sly syndrome).

MPS VII is a progressive, rare, genetic disorder that effects an estimated 200 people in the developed world. People with MPS VII do not produce enough beta-glucuronidase, an enzyme that plays a key part in the breakdown of specific sugars in the body. The lack of this enzyme leads to storage of sugars within the cells, which can cause progressive damage in many organs.

INDICATION (IN THE E.U.)

Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). For more detail, including complete Summary of Product Characteristics (SmPC), please visit https://www.ultragenyx.com/wp-content/uploads/2021/09/SmPC_Mepsevii_INN-vestronidase_alfa_IT.pdf.

IMPORTANT SAFETY INFORMATION (IN THE E.U.)Contraindications: Life-threatening hypersensitivity (anaphylactic reaction) to the active substance or to any of the excipients.

Special warnings and precautions for use: The effects of treatment with vestronidase alfa should be periodically evaluated and discontinuation of treatment should be considered in cases where clear benefits (including stabilisation of disease manifestations) are not observed. Discontinuation of treatment may cause significant worsening of the patient’s clinical status. As end organ damage progresses over time, it is more difficult for the treatment to reverse the damage or to show improvements. It should be considered by the treating physician that the administration of vestronidase alfa does not affect the irreversible complications (e.g. skeletal deformities). Vestronidase alfa, at the exposure observed in humans, is not expected to cross the blood-brain-barrier and therefore it is not likely to impact the neurological manifestations of the disease. Hypersensitivity reactions including anaphylaxis: Serious hypersensitivity reactions, including anaphylaxis, have been reported with vestronidase alfa; appropriate medical support should be readily available when vestronidase alfa is administered. Infusion should be avoided if the patient has an acute febrile or respiratory illness at the time. Spinal/Cervical cord compression: Spinal or cervical cord compression is a known and serious complication of MPS VII. Patients with MPS VII receiving vestronidase alfa should be monitored for signs and symptoms of spinal cord compression or neck instability including neck or back pain, weakness of limbs, changes in reflexes or urinary and faecal incontinence. Appropriate clinical treatment should be immediately sought. Sodium restricted diet: Mepsevii is considered high in sodium. This should be taken into consideration during dilution of the medicinal product for patients on a controlled sodium diet or for those patients with congestive heart failure needing to restrict sodium and total water intake. Traceability: the name and the batch number of the administered product should be clearly recorded.

Undesirable effects: The most common adverse reactions from 4 clinical trials in 23 patients treated with vestronidase alfa were rash (17.4%), urticaria (17.4%), infusion site extravasation (17.4%), anaphylactoid reaction (13%), infusion site swelling (8.7%), pruritus (8.7%) and diarrhoea (8.7%). Most adverse reactions were mild to moderate in severity. There was a single adverse reaction of febrile convulsion observed in one patient (4.3%); the patient recovered without sequelae. See the SmPC for further information.

About Ultragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

Forward-Looking Statements

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the company’s commercialization activities, business and operating results, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, uncertainties related to insurance coverage and reimbursement status of the company’s newly approved products, the company’s evolving integrated commercial organization and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, and the availability or commercial potential of Ultragenyx’s products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 3, 2021, and its subsequent periodic reports filed with the Securities and Exchange Commission.

ContactsUltragenyx Pharmaceutical Inc.MediaCarolyn WangIR@ultragenyx.com

Ultragenyx ItalyClaudio Angelinicangelini@ultragenyx.com+39 331 6549412