Sensorion receives Orphan Drug Designation for OTOF-GT for the treatment of otoferlin gene-mediated hearing loss from the US Food and Drug Administration
30 Novembre 2022 - 7:30AM
Business Wire
- Designation follows recent FDA award of Rare Pediatric Disease
Designation to Sensorion for OTOF-GT program
- Sensorion is on track to file a CTA for OTOF-GT in H1 2023
Regulatory News:
Sensorion (FR0012596468 – ALSEN) a pioneering
clinical-stage biotechnology company which specializes in the
development of novel therapies to restore, treat and prevent within
the field of hearing loss disorders, announces that the US Food and
Drug Administration (FDA) has granted Orphan Drug Designation (ODD)
to OTOF-GT, Sensorion’s lead gene therapy program, intended for the
treatment of otoferlin gene mediated hearing loss. Sensorion is on
track to file a Clinical Trial Application for OTOF-GT in the first
half of 2023.
Sensorion’s OTOF-GT dual vector AAV gene therapy development
program aims to restore hearing in people living with otoferlin
deficiency. Patients with mutations in OTOF suffer from severe to
profound sensorineural prelingual non syndromic hearing loss.
Otoferlin deficiency is responsible for up to 8% of all cases of
congenital hearing loss, with around 20,000 people affected in the
US and Europe1.
Sensorion has progressed preclinical and clinical development
plans for OTOF-GT and is on track to file a Clinical Trial
Application (CTA) in the first half of 2023. In September 2022, the
Company received a positive opinion on an application for Orphan
Drug Designation (ODD) from the European Medicines Agency (EMA).
The European Commission issued the decision on October 11, 2022. On
November 7, 2022, Sensorion has also been granted Rare Pediatric
Disease Designation to OTOF-GT by the US FDA.
“We are really pleased to have received this significant
regulatory feedback from the FDA, following the agency’s recent
award of Rare Pediatric Disease designation for OTOF-GT. Orphan
Drug Designation will support us in advancing our development
program, a gene therapy which offers the potential to help patients
with a condition for which there are currently no approved curative
therapies,” said Géraldine Honnet, Chief Medical Officer of
Sensorion. “We are excited to have achieved this milestone and
remain highly focused on the development of our most promising
candidates to produce life-changing therapies to restore, treat and
prevent hearing loss disorders.”
Orphan Drug Designation is granted by the FDA to encourage
development of treatment, diagnosis, or prevention of rare
diseases, defined as those affecting fewer than 200,000 people in
the US. The designation affords Sensorion the potential for certain
benefits, including up to seven years of post-approval market
exclusivity, assistance in the drug development process, tax
credits for clinical development, and exemptions for certain FDA
fees.
Under Rare Pediatric Disease designation, a sponsor who receives
an approval for a drug or biologic for a “rare pediatric disease”
may be eligible for a voucher that can be redeemed to receive
priority review of a subsequent marketing application for a
different product or sold to another sponsor for priority review of
their marketing application, an opportunity for which there is a
robust market.
About Sensorion
Sensorion is a pioneering clinical-stage biotech company, which
specializes in the development of novel therapies to restore, treat
and prevent hearing loss disorders, a significant global unmet
medical need.
Sensorion has built a unique R&D technology platform to
expand its understanding of the pathophysiology and etiology of
inner ear related diseases, enabling it to select the best targets
and mechanisms of action for drug candidates. Its portfolio
combines both small molecule programs and a preclinical portfolio
of inner ear gene therapies.
Its clinical-stage portfolio includes one Phase 2 product:
SENS-401 (Arazasetron) progressing in a planned Phase 2 proof of
concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity
(CIO) and, with partner Cochlear Limited, in a study of SENS-401 in
patients scheduled for cochlear implantation. A Phase 2 study of
SENS-401 was also completed in Sudden Sensorineural Hearing Loss
(SSNHL) in January 2022.
Sensorion pursues its broad strategic collaboration with
Institut Pasteur focused on the genetics of hearing. It has two
gene therapy programs aimed at correcting hereditary monogenic
forms of deafness including OTOF-GT, targeting deafness caused by a
mutation of the gene encoding for otoferlin, and hearing loss
related to mutation in GJB2 gene to potentially address important
hearing loss segments in adults and children (GJB2-GT). The Company
is also working on the identification of biomarkers to improve
diagnosis of these underserved illnesses.
www.sensorion.com
Label: SENSORION
ISIN: FR0012596468
Mnemonic: ALSEN
Disclaimer
This press release contains certain forward-looking statements
concerning Sensorion and its business. Such forward looking
statements are based on assumptions that Sensorion considers to be
reasonable. However, there can be no assurance that such
forward-looking statements will be verified, which statements are
subject to numerous risks, including the risks set forth in the
2021 full year financial report published on April 28, 2022, and
available on our website and to the development of economic
conditions, financial markets and the markets in which Sensorion
operates. The forward-looking statements contained in this press
release are also subject to risks not yet known to Sensorion or not
currently considered material by Sensorion. The occurrence of all
or part of such risks could cause actual results, financial
conditions, performance or achievements of Sensorion to be
materially different from such forward-looking statements. This
press release and the information that it contains do not
constitute an offer to sell or subscribe for, or a solicitation of
an offer to purchase or subscribe for, Sensorion shares in any
country. The communication of this press release in certain
countries may constitute a violation of local laws and regulations.
Any recipient of this press release must inform oneself of any such
local restrictions and comply therewith.
1 Rodríguez-Ballesteros M, Reynoso R, Olarte M, Villamar M,
Morera C, Santarelli R, Arslan E, Medá C, Curet C, V�lter C,
Sainz-Quevedo M, Castorina P, Ambrosetti U, Berrettini S, Frei K,
Tedín S, Smith J, Cruz Tapia M, Cavallé L, Gelvez N, Primignani P,
Gómez-Rosas E, Martín M, Moreno-Pelayo MA, Tamayo M, Moreno-Barral
J, Moreno F, del Castillo I. A multicenter study on the prevalence
and spectrum of mutations in the otoferlin gene (OTOF) in subjects
with nonsyndromic hearing impairment and auditory neuropathy. Hum
Mutat. 2008 Jun;29(6):823-31. doi: 10.1002/humu.20708. PMID:
18381613.
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Investor Relations Noemie Djokovic Investor Relations and
Communications +33 6 76 67 98 31
ir.contact@sensorion-pharma.com
International Media Relations Consilium Strategic
Communications Matthew Cole/Jessica Hodgson +44 7593 572720 +44
7561 424788 Sensorion@consilium-comms.com
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