US FDA approves Ipsen’s SohonosTM (palovarotene) capsules, the
first and only treatment for people with fibrodysplasia ossificans
progressiva
- Breakthrough treatment reduces new, abnormal bone formation in
soft and connective tissues, in people living with ultra-rare bone
disease, fibrodysplasia ossificans progressiva (FOP)
- FOP impacts about 400 people in the U.S., it leads to
progressive mobility loss, severely limits quality of life and
shortens median life expectancy to 56 years
- SohonosTM may be prescribed immediately in the U.S. for
eligible patients, aged 8 years and older for females and 10 years
and older for males
PARIS, FRANCE, 16 August 2023 –
Ipsen (Euronext: IPN; ADR: IPSEY) announced today approval by the
U.S. Food and Drug Administration (FDA) of Sohonos™ (palovarotene)
capsules as a retinoid indicated for the reduction in volume of new
heterotopic ossification in adults and pediatric patients aged 8
years and older for females and 10 years and older for males with
fibrodysplasia ossificans progressiva (FOP).
“The FDA approval of Sohonos is a breakthrough
for the U.S. FOP community. For the first time doctors have an
approved medicine available to them, shown to reduce the formation
of new, abnormal bone growth, known as heterotopic ossification
(HO), which causes debilitating mobility challenges and has a
devastating impact on the lives of people with FOP,” said Howard
Mayer, Head of Research and Development, Ipsen. “Development of
medicines for rare diseases takes commitment and belief from
everyone involved. We at Ipsen are sincerely grateful to the FOP
community of patients and medical experts, as the first-ever
treatment in the U.S. for managing FOP would not be possible
without their participation in the clinical trials and ongoing
support.”
FOP impacts the lives of an estimated 400 people
in the U.S. and 900 people globally.1,2 As the disease continuously
progresses with flare-up episodes causing rapid bone growth, HO
severely restricts mobility and function.3,4 Most people living
with FOP inevitably lose the ability to eat and drink on their own,
cannot provide selfcare or use the restroom themselves, and are
unable to maintain employment.5 By the age of 30 years old, the
majority of people with FOP require a wheelchair and full-time
caregiver assistance.3,6 The management of FOP has previously been
limited to palliative care and ultimately, FOP shortens the median
life expectancy to 56 years, untimely death is often caused by bone
formation around the ribcage leading to breathing problems and
cardiorespiratory failure, or falls resulting in fractures or head
injuries because joint ankylosis prevents bracing from a fall.7
“FOP is life-altering to the individuals
diagnosed and their families. There’s not a day that goes by where
those impacted don’t worry about the debilitating physical pain of
muscle that is replaced by bone, another joint locking or the
relentless emotional toll of losing the ability to do an activity
they love, or hold a loved one close," explained Michelle Davis,
Executive Director of International FOP Association. "The first
treatment for FOP has been proven to reduce the volume of new
abnormal bone growth, which may result in better health outcomes
for people living with FOP.”
The FDA approval is based on the pivotal
efficacy and safety data from the Phase III MOVE trial, the first
and largest multicenter, open-label trial in adult and pediatric
patients. The 18-month data published in the Journal of Bone and
Mineral Research,8 included 107 patients (12 percent of the
estimated number of individuals worldwide living with FOP) who
received oral palovarotene compared with untreated individuals from
Ipsen’s global FOP Natural History Study.9 The study results
demonstrated palovarotene effectively reduced annualized
heterotopic ossification volume compared with no treatment beyond
standard of care, (54% reduction with weighted linear mixed effect
model).8 The study also demonstrated that palovarotene has a
well-characterized safety profile, with adverse events consistent
with the systemic retinoid class. The most common treatment
emergent adverse reactions reported in the study were mucocutaneous
events such as dry skin, lip dryness, alopecia, drug eruption,
rash, and pruritus and musculoskeletal events such as arthralgia
and premature growth plate closure in growing children. 8
“As a clinician caring for patients with FOP, I
personally see the daily challenges and stresses that our patients
and their families must contend with,” said Dr Edward Hsiao,
Professor of Medicine, Division of Endocrinology and Metabolism,
University of California, San Francisco. “The published Phase III
MOVE study showed that Sohonos can decrease new heterotopic
ossification, and that palovarotene can be tolerated by many
patients with FOP. Sohonos is not for everyone. As with all
medicines there are risks in this case especially for young
children who may develop early growth plate closure. In addition,
Sohonos has the same side effects as other retinoids, including
dryness of the skin and mucus membranes. However, since the
accumulation of HO in FOP is progressive, irreversible, and life
altering, this medication is an important treatment option for our
FOP community.”
Sohonos, the first and
only treatment for FOP
Sohonos is an oral medicine with particular
selectivity for the gamma subtype of retinoic-acid receptors, which
are an important regulator of skeletal development and ectopic bone
in the retinoid signaling pathway. The medicine is designed to
mediate the interactions between the receptors, growth factors and
proteins within the retinoid signaling pathway to reduce new
abnormal bone formation in FOP. The recommended dosing for Sohonos
includes a chronic daily dosage of 5 mg (or weight-based equivalent
for pediatric patients under 14 years of age), which can be
modified/increased for flare-up symptoms. Sohonos may be prescribed
immediately in the U.S. for eligible patients.
To ensure access to Sohonos for eligible
individuals in the U.S., Ipsen Cares patient support program is
available as a resource to people living with FOP and their
caregivers to provide educational support and address coverage,
access and reimbursement questions (1-866-435-5677).
Sohonos received Orphan Drug and Breakthrough
Therapy Designations from the U.S. Food and Drug Administration
(FDA) for the treatment of FOP and was granted Priority Review of
the New Drug Application (NDA). Sohonos, under the generic name
palovarotene, is also under review with a number of other
regulatory authorities. In July 2023, the European Commission did
not grant marketing authorization for palovarotene. SohonosTM
(palovarotene capsules) is currently authorized for use in eligible
patients in U.S., Canada,10 and with a conditional approval in the
United Arab Emirates.
With this approval the FDA also issued a Rare
Pediatric Disease Priority Review Voucher (PRV). The voucher can be
used for subsequent drug applications that would not qualify for a
priority review.
Important
Sohonos Safety
Information
WARNING: EMBRYO-FETAL TOXICITY and PREMATURE PHYSEAL
CLOSURE IN GROWING PEDIATRIC PATIENTS
Embryo-Fetal ToxicitySOHONOS is
contraindicated in pregnancy. SOHONOS can cause fetal harm. Because
of the risk of teratogenicity and to minimize fetal exposure,
SOHONOS is to be administered only if conditions for pregnancy
prevention are met.
Premature
Epiphyseal
ClosurePremature
epiphyseal closure occurs in growing pediatric patients treated
with SOHONOS, close monitoring is
recommended. |
ContraindicationsSOHONOS is contraindicated in
patients during pregnancy, or with a history of allergy or
hypersensitivity to retinoids, or to any component of SOHONOS.
Warnings and Precautions
- Embryo-Fetal
Toxicity: SOHONOS can cause fetal harm and is
contraindicated during pregnancy. Advise females of reproductive
potential to use an effective method of contraception during
treatment with SOHONOS and for 1 month after the last dose. If a
pregnancy occurs during Sohonos treatment, discontinue treatment
immediately and refer the patient to an obstetrician/gynecologist
experienced in reproductive toxicity.
- Premature Epiphyseal
Closure in Growing Pediatric Patients: SOHONOS can cause
irreversible premature epiphyseal closure and potential adverse
effects on growth. Prior to starting treatment with SOHONOS, all
growing pediatric patients should undergo baseline assessment of
skeletal maturity and continued monitoring until patients reach
skeletal maturity or final adult height. If appropriate, temporary
or permanent discontinuation may be warranted.
- Mucocutaneous Adverse
Reactions: Dry skin, lip dry, pruritis,
rash, alopecia, erythema, skin exfoliation [skin peeling] and, dry
eye occurred with SOHONOS. Prophylactic measures to minimize risk
and/or treat the mucocutaneous adverse reactions are recommended
(e.g., skin emollients, sunscreen, lip moisturizers, or artificial
tears). Some may require dose reduction or discontinuation.
Photosensitivity reactions have been associated with the use of
retinoids and may occur with SOHONOS. Precautionary measures for
phototoxicity are recommended (e.g., use of sunscreens, protective
clothing, and use of sunglasses).
- Metabolic Bone
Disorders: Increased risk of radiologically observed
vertebral fractures and decreased vertebral bone mineral content
and bone density. Periodic radiological assessment of the spine is
recommended. Retinoids have been associated with hyperostotic
changes (bone spurs) and calcification of tendons or ligaments may
occur with SOHONOS.
- Psychiatric
Disorders: New or worsening psychiatric events were
reported with SOHONOS including depression, anxiety, mood
alterations, and suicidal thoughts and behaviors. Monitor for
development of new or worsening psychiatric symptoms during
treatment with SOHONOS. Patients and/or caregivers should contact
their healthcare provider if new or worsening psychiatric symptoms
develop during treatment with SOHONOS.
- Night Blindness:
This may be dose-dependent, making driving a vehicle at night
potentially hazardous during treatment. Advise patients to be
cautious when driving or operating any vehicle at night and seek
medical attention in the event of vision impairment.
Adverse Reactions
The most common adverse reactions (≥ 10%) in
clinical trials include dry skin, lip dry, arthralgia, pruritus,
pain in extremity, rash, alopecia, erythema, headache, back pain,
skin exfoliation [skin peeling] , nausea, musculoskeletal pain,
myalgia, dry eye, hypersensitivity, peripheral edema, and
fatigue.
Drug Interactions
- CYP3A4 inhibitors may increase
SOHONOS exposure. Avoid concomitant use of strong or moderate
CYP3A4 inhibitors, as well as grapefruit, pomelo or juices
containing these fruits.
- CYP3A4 inducers may decrease
SOHONOS exposure. Avoid concomitant use of strong or moderate
CYP3A3 inducers.
- The use of both vitamin A and
SOHONOS at the same time may lead to additive effects. Concomitant
administration of vitamin A in doses higher than the recommended
daily allowance and/or other oral retinoids must be avoided due to
risk of hypervitaminosis A.
- Systemic retinoid use has been
associated with cases of benign intracranial hypertension
(pseudotumor cerebri), some of which involved the concomitant use
of tetracyclines. Avoid coadministration of SOHONOS with
tetracycline derivatives.
Use in Specific Populations
- Pregnancy: SOHONOS
is contraindicated during pregnancy. Obtain a negative serum
pregnancy test within 1 week prior to SOHONOS therapy and
periodically, as needed, over the course of treatment with SOHONOS
and 1 month after treatment discontinuation unless patient is not
at risk of pregnancy. If pregnancy occurs during treatment with
SOHONOS, stop treatment immediately and refer the patient to an
obstetrician/gynecologist or other specialist experienced in
reproductive toxicity for evaluation and advice.
- Lactation: Advise
females that breastfeeding is not recommended during treatment with
SOHONOS, and for at least 1 month after the last dose.
- Females and Males of
Reproductive Potential: Advise females of reproductive
potential to use effective contraception at least 1 month prior to
and during treatment, and for 1 month after the last dose unless
continuous abstinence is chosen.
- Pediatric Use: All
growing pediatric patients should undergo baseline assessment of
growth and skeletal maturity before starting treatment and
continued clinical and radiographic monitoring every 6-12 months
until patients reach skeletal maturity or final adult height
- Renal or Hepatic
Impairment: Use of SOHONOS in patients with severe renal
impairment, or with moderate or severe hepatic impairment is not
recommended.
Please see full Prescribing Information, including BOXED WARNING
on Ipsen.com/us
ENDS
About Ipsen Ipsen is a
global, mid-sized biopharmaceutical company focused on
transformative medicines in Oncology, Rare Disease and
Neuroscience. With total sales of €3.0bn in FY 2022, Ipsen sells
medicines in over 100 countries. Alongside its
external-innovation strategy, the Company’s research and
development efforts are focused on its innovative and
differentiated technological platforms located in the heart of
leading biotechnological and life-science hubs: Paris-Saclay,
France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has
around 5,300 colleagues worldwide and is listed in Paris (Euronext:
IPN) and in the U.S. through a Sponsored Level I American
Depositary Receipt program (ADR: IPSEY). For more information,
visit ipsen.com
For further information:
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1 Pignolo RJ, et al Prevalence of fibrodysplasia ossificans
progressiva (FOP) in the United States: estimate from three
treatment centers and a patient organisation. Orphanet J Rare Dis
(2021) 16 : 3502 Baujat G, et al. Prevalence of
fibrodysplassia ossificans progressiva (FOP) in France: an estimate
based on a record linkage of two national databases. Orphanet J
Rare Dis (2017) 12: 1233 Pignolo RJ, et al. Self-reported baseline
phenotypes from the International Fibrodysplasia Ossificans
Progressiva (FOP) Association Global Registry. Bone
2020;134:115274.4 Pignolo et al. The Natural History of
Fibrodysplasia Ossificans Progressiva: A Prospective, Global,
36-Month Study. Genetics in Medicine. 2022.
https://doi.org/10.1016/j.gim.2022.08.0135. Al Mukaddam M, et al.
Val Health 2022;25:S273 (POSA427)6 Pignolo RJ, et al.
Fibrodysplasia ossificans progressiva: diagnosis, management, and
therapeutic horizons. Pediatr Endocrinol Rev 2013;10 Suppl 2:437–
48.7 Kaplan et al, Early Mortality and Cardiorespiratory Failure in
Patients with Fibrodysplasia Ossificans Progressiva. The Journal of
Bone & Joint Surgery. 2010. doi: 10.2106/JBJS.I.007058
Pignolo RJ, et al. Reduction of New HO in the Open-Label, Phase 3
MOVE Trial of Palovarotene for Fibrodysplasia Ossificans
Progressiva (FOP). J Bone Miner Res. 2022.9. Pignolo RJ, et al. The
natural history of fibrodysplasia ossificans progressiva: A
prospective 36-month study. Gen Med. 2022,ISSN
1098-3600,https://doi.org/10.1016/j.gim.2022.08.013.10. Government
of Canada, Notice: Multiple Additions to the Prescription Drug List
(PDL). Viewed 30 November 2022,
<https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/prescription-drug-list/notices-changes/multiple-additions-2022-01-24.html>.
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