Press Release: Novartis announces landmark EU approval for one-time gene therapy Luxturna(R) to restore vision in people with rare inherited retinal disease

Novartis International AG / Novartis announces landmark EU approval for

one-time gene therapy Luxturna(R) to restore vision in people with rare

inherited retinal disease. Processed and transmitted by West

Corporation. The issuer is solely responsible for the content of this

announcement.

-- Luxturna* (voretigene neparvovec) is the first gene therapy to treat an

inherited retinal disease, indicated for children and adults with vision

loss caused by mutations in both copies of the RPE65 gene and sufficient

viable retinal cells[1]

-- Nearly 60% of patients have severe forms of the disease, with severe

visual impairment occurring shortly after birth[2]

-- As a one-time treatment, Luxturna restores vision and improves sight in

children and adults with a sustained effect and favorable safety

profile[3],[4]

The digital press release with multimedia content can be accessed here:

https://novartis.gcs-web.com/Novartis-announces-landmark-EU-approval-for-one-time-gene-therapy-Luxturna-to-restore-vision-in-people-with-rare-inherited-retinal-disease

Basel, November 23, 2018 - Novartis announced today that the European

Commission (EC) approved Luxturna, a one-time gene therapy for the

treatment of patients with vision loss due to a genetic mutation in both

copies of the RPE65 gene and who have enough viable retinal cells. The

authorization is valid in all 28 member states of the EU, as well as

Iceland, Liechtenstein and Norway. Luxturna was developed and is

commercialized in the US by Spark Therapeutics.

People born with mutations in both copies of the RPE65 gene can

experience profound sight loss from an early age, with the majority of

patients progressing to total blindness[3]. Research shows that vision

impairment and blindness in children frequently cause social isolation,

emotional distress, loss of independence, or hazards such as falls and

injuries[5]. The working copy of the RPE65 gene provided by Luxturna can

restore vision and improve sight in children and adults with sufficient

viable retinal cells[3].

"Today's approval is momentous for patients given that there have been

no pharmacological treatment options to date to treat this form of LCA,"

said Christina Fasser, president of Retina International, an umbrella

organization of more than 43 patient organizations worldwide promoting

research to find treatments for inherited retinal degenerative diseases.

"Access to this treatment has the potential to reduce the substantial

physical, emotional and financial burden this disease has on patients

and their families."

"As a clinician who has worked for over 20 years with patients with

inherited retinal disease and their families, I've seen firsthand the

profound impact blindness can have on quality of life. It's exciting to

practice medicine at a time when we can offer options to children and

adults facing blindness," said Dr Bart Leroy, ophthalmologist and

clinical geneticist, Professor and Head, Department of Ophthalmology at

Ghent University Hospital, Ghent, Belgium and Director of Retinal

Degenerations Clinic at Children's Hospital of Philadelphia,

Philadelphia, PA. "After more than 20 years of gene therapy research,

there is finally a promising future ahead for the treatment of rare

genetic eye disorders."

"EU approval of the one-time gene therapy Luxturna marks a milestone in

reimagining medicine and can bring real value to patients, their

families and society as a whole," said Paul Hudson, CEO, Novartis

Pharmaceuticals. "Novartis is committed to working with patients,

caregivers, health systems and physicians to establish access to this

gene therapy for RPE65 patients, as we believe it can help restore sight

and improve vision in children and adults who currently have no

treatment options."

The EC decision is based on a positive CHMP opinion that looked at data

from a Phase 1 clinical trial, its follow-up trial, and the first

randomized, controlled Phase 3 gene therapy trial for an inherited

disease[3]. In the Phase 3 clinical trial vision improvement was

recorded as early as 30 days following treatment[3]. At 1 year, compared

to the control group, patients treated with voretigene neparvovec

improved by 1.6 light levels on the binocular multi-luminance mobility

test (MLMT), the trial's novel, patient-centric, primary endpoint[3].

Vision improved by one or more light levels for 90% of patients treated

with voretigene neparvovec, and 65% were able to successfully navigate

the MLMT at the lowest light level of 1 lux at 1 year[3].

Decisions from national reimbursement bodies on Luxturna for patients

with vision loss due to a genetic mutation in both copies of the RPE65

gene are expected in 2019 and 2020. Novartis is working closely with all

stakeholders to help ensure that eligible patients can start benefitting

from this treatment as quickly as possible once reimbursement decisions

are available in 2019 and 2020. A range of resources and innovative

reimbursement and access approaches are also being explored in order to

provide as much support as possible to both patients and healthcare

professionals.

About RPE65 mutation-associated inherited retinal disease

Inherited retinal diseases are a group of rare blinding conditions

caused by more than 250 different genes[6], often disproportionally

affecting children and young adults[3]. Mutations in both copies of the

RPE65 gene affect approximately 1 in 200,000 people[7].

Mutations in both copies of the RPE65 gene can lead to blindness. Early

in the disease patients can suffer from night blindness (nyctalopia),

loss of light sensitivity, loss of peripheral vision, loss of sharpness

or clarity of vision, impaired dark adaptation and repetitive

uncontrolled movements of the eye (nystagmus)[8]. Patients with

mutations in both copies of the RPE65 gene may be diagnosed, for

instance, with subtypes of either Leber congenital amaurosis or

retinitis pigmentosa[9]. A genetic test is needed to confirm that vision

loss is caused by mutations in the RPE65 gene[3].

About the multi-luminance mobility test (MLMT)

The MLMT measures changes in patient relevant functional vision by

asking patients to navigate a course accurately and at a reasonable pace

at seven different levels of illumination, ranging from 400 lux

(corresponding to a brightly lit office) to one lux (corresponding to a

moonless summer night)[3].

About Luxturna's mechanism of action

Luxturna is the first EU-approved treatment for this disease and is

designed to provide a working copy of the RPE65 gene to act in place of

the mutated RPE65 gene[3].

About the Novartis and Spark Therapeutics licensing and supply agreement

In January 2018, Spark Therapeutics entered into a licensing and supply

agreement with Novartis covering development, registration and

commercialization rights to Luxturna in markets outside the US. Upon the

transfer of the marketing authorization from Spark Therapeutics to

Novartis, Novartis can commercialize Luxturna in the EU/EEA. Novartis

already has exclusive rights to pursue development, registration and

commercialization in all other countries outside the US, and Spark

Therapeutics will supply the gene therapy to Novartis.

About Novartis in ophthalmology

For more than 70 years, patients, caregivers and healthcare providers

worldwide have looked to Novartis for state-of-the-art treatments in eye

diseases. We continue to invest in science as well as in strategic

alliances to help ensure patients have access to screening, diagnosis,

and our eye medicines. Our commitment to vision extends globally across

ages, from premature infants to seniors, from rare diseases to those

affecting millions, from eye drops to gene therapies. Our aspiration:

reimagining eye care to help everyone see possibilities.

Disclaimer

This press release contains forward-looking statements within the

meaning of the United States Private Securities Litigation Reform Act of

1995. Forward-looking statements can generally be identified by words

such as "potential," "can," "will," "plan," "expect," "anticipate,"

"look forward," "believe," "committed," "investigational," "pipeline,"

"launch," or similar terms, or by express or implied discussions

regarding potential marketing approvals, new indications or labeling for

the investigational or approved products described in this press release,

or regarding potential future revenues from such products. You should

not place undue reliance on these statements. Such forward-looking

statements are based on our current beliefs and expectations regarding

future events, and are subject to significant known and unknown risks

and uncertainties. Should one or more of these risks or uncertainties

materialize, or should underlying assumptions prove incorrect, actual

results may vary materially from those set forth in the forward-looking

statements. There can be no guarantee that the investigational or

approved products described in this press release will be submitted or

approved for sale or for any additional indications or labeling in any

market, or at any particular time. Nor can there be any guarantee that

such products will be commercially successful in the future. In

particular, our expectations regarding such products could be affected

by, among other things, the uncertainties inherent in research and

development, including clinical trial results and additional analysis of

existing clinical data; regulatory actions or delays or government

regulation generally; global trends toward health care cost containment,

including government, payor and general public pricing and reimbursement

pressures; our ability to obtain or maintain proprietary intellectual

property protection; the particular prescribing preferences of

physicians and patients; general political and economic conditions;

safety, quality or manufacturing issues; potential or actual data

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November 23, 2018 01:15 ET (06:15 GMT)