Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical
company focused on the development of novel products for serious
rare and ultra-rare genetic diseases, today announced that Brazil’s
National Health Surveillance Agency (ANVISA) has approved Dojolvi®
(triheptanoin) as a source of calories and fatty acids for the
treatment of pediatric and adult patients with molecularly
confirmed long-chain fatty acid oxidation disorders (LC-FAOD).
“Patients with LC-FAOD face frequent
hospitalizations and major medical events with limited options to
support management of their disease,” said Eduardo Thompson, Senior
Vice President and Regional Head, Latin America at Ultragenyx.
“This approval of Dojolvi is critical to providing therapeutic
options for patients in Brazil and we are now working with urgency
to obtain reimbursement approval in order to support broad access
to therapy. We are grateful to the community of patients,
caregivers and physicians whose efforts have contributed to this
important approval.”
Dojolvi is also approved by the U.S. Food and Drug
Administration (FDA) and by Health Canada for the treatment of
adult and pediatric patients with LC-FAOD. Outside of the U.S.,
patients have had access to Dojolvi through named patient and early
access programs.
About LC-FAODLC-FAOD are a group
of autosomal recessive genetic disorders characterized by metabolic
deficiencies in which the body is unable to convert long-chain
fatty acids into energy. The inability to produce energy from fat
can lead to severe depletion of glucose in the body and serious
complications, which can lead to hospitalizations or early death.
Other current treatment options for LC-FAOD include avoidance of
fasting, low-fat/high-carbohydrate diets, carnitine and even-carbon
medium-chain triglyceride (MCT) oil, a medical food product.
LC-FAOD affect an estimated 8,000 to 14,000
children and adults in the developed world and are included in
newborn screening panels in various geographies due to the risk for
serious outcomes including death early in life. The Brazilian
government recently sanctioned law 14.154/2021 that expands the
list of diseases to be included in the National Newborn Screening
Program carried out in the Unified Health System (SUS), including
LC-FAOD. The implementation will take place in five stages within a
period to be regulated by the Ministry of Health, with LC-FAOD
included at the second stage.
About DojolviDojolvi is a highly
purified, pharmaceutical-grade, odd-carbon medium-chain
triglyceride consisting of three 7-carbon fatty acids on a glycerol
backbone created via a multi-step chemical process. It is designed
to provide medium-chain, odd-carbon fatty acids as an energy source
and metabolite replacement for people with LC-FAOD.
INDICATIONDojolvi is indicated as
a source of calories and fatty acids for the treatment of pediatric
and adult patients with molecularly confirmed long-chain fatty acid
oxidation disorders (LC-FAOD).
IMPORTANT SAFETY
INFORMATIONWARNINGS AND
PRECAUTIONSFeeding Tube Dysfunction
- Feeding tube dysfunction was reported in patients receiving
triheptanoin. The contribution of Dojolvi cannot be ruled out.
Dojolvi should not be administered in feeding tubes manufactured of
polyvinyl chloride (PVC). The feeding tube should be monitored
regularly to ensure proper functioning and integrity.
Intestinal Malabsorption in Patients with
Pancreatic Insufficiency
- Low or absent pancreatic enzymes may result in reduced
absorption of heptanoate subsequently leading to insufficient
supplementation of medium-chain fatty acids. Administration of
Dojolvi should be avoided in patients with pancreatic
insufficiency.
ADVERSE
REACTIONSGastrointestinal (GI)
- The most common GI-related adverse reactions reported in the
pooled safety population of Studies 1 and 2 were abdominal pain
(abdominal discomfort, abdominal pain, abdominal distension,
abdominal pain upper, GI pain) [60%], diarrhea [44%], vomiting
[44%], and nausea [14%].
DRUG
INTERACTIONSPancreatic Lipase
Inhibitors
- Co-administration should be avoided due to potential for
reduced clinical effect of Dojolvi.
USE IN SPECIFIC
POPULATIONSPregnancy and Lactation
- There are no available data on triheptanoin use in pregnant
women to evaluate for a drug-associated risk of major birth
defects, miscarriage, or adverse maternal or fetal outcomes. Women
are advised to report pregnancies to Ultragenyx Pharmaceutical Inc.
at 1-888-756-8657 (U.S.) or 0800-770-4481 (Brazil).
- There are no data on the presence of triheptanoin or its
metabolites in human or animal milk, the effects on the breastfed
infant, or the effects on milk production.
PATIENT COUNSELING
INFORMATIONThe patient or caregiver
should:
- Read the FDA/ANVISA-approved patient labeling, which includes
information on the appropriate oral or feeding tube preparation,
administration, and storage.
- Regularly inspect the feeding tube for proper functioning and
integrity and report to the healthcare provider if any issues are
identified.
- Be informed that pancreatic insufficiency may reduce the
clinical effect of Dojolvi and to report any known pancreatic
insufficiency to the healthcare provider.
In Brazil, you may report side effects to
Ultragenyx Pharmaceutical Inc. using the local toll-free number at
0800-770-4481 or by email at ultragenyx@primevigilance.com, or FDA
at 1-800-FDA-1088 or www.fda.gov/medwatch
Please see Prescribing Information for physicians
and the patient package insert (both in Portuguese) for a complete
discussion of the risks associated with Dojolvi. The texts are
based on the U.S. Prescribing Information and the indication is the
same.
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, its commercialization,
marketing and manufacturing capabilities and strategy 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 our 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.Investors Joshua Higa415-660-0951
Media Carolyn Wang415-225-5050
Ultragenyx Brazil Offices0800-770-4481Vitor
Brandao, Regulatory Affairs Director
Brazilvbrandao@ultragenyx.comRua Josefina, 200, Guarulhos Sao
Paulo, 07093-080, Brazil
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