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The Development Timeline of LUXTURNA® (Voretigene Neparvovec-Rzyl)

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The Development Timeline of LUXTURNA® (Voretigene Neparvovec-Rzyl) Striving to challenge the inevitability of genetic disease by discovering, developing and delivering treatments in ways unimaginable – until now. The development timeline of LUXTURNA® (voretigene neparvovec-rzyl) Apr. Orphan drug designation received from the European Medicines 2012 Agency (EMA) for Leber congenital amaurosis (LCA) Nov. 2012 Pivotal Phase 3 clinical trial initiated Mar. Spark 2013 Therapeutics founded Nov. Breakthrough therapy designation 2014 received from U.S. FDA Announcement of data from first completed, randomized Oct. and controlled Phase 3 trial 2015 of a gene therapy for a genetic disease Jul. Safety and ecacy data 2016 in contralateral eye from a Phase 1 study published in The Lancet Two-year ecacy and safety data Oct. from Phase 3 study presented at the American Academy of Ophthalmology 2016 Annual Meeting 2016 Jan. Four-year ecacy and safety data 2017 announced from a Phase 1 follow-on study Jan. Amended FDA orphan drug designation announced for “the treatment of inherited 2017 retinal dystrophy due to biallelic RPE65 mutations” Pivotal Phase 3 clinical trial Jul. data published in The Lancet 2017 FDA accepts for filing Jul. Biologics License Application (BLA) and grants 2017 Priority Review with Prescription Drug User Fee Act (PDUFA) date of January 12, 2018 EMA validates Aug. submitted Marketing Authorization Application (MAA) 2017 Aug. Study published in Clinical and Experimental Ophthalmology confirming 2017 multi-luminance mobility test's construct and content, validity, reliability and ability to detect change in functional vision FDA advisory committee unanimously (16-0) votes in favor of benefit-risk profile. The vote is non-binding, Oct. but FDA takes the advisory committee's recommendation into consideration when 2017 reviewing the Biologics License Application (BLA) Dec. FDA approves LUXTURNA® (voretigene neparvovec-rzyl), the first gene 2017 therapy for a genetic disease in the U.S. EU approval Nov. LUXTURNA® (voretigene neparvovec) becomes first gene therapy for a genetic 2018 disease to be approved in both the U.S. and EU. For more information please go to your country’s Novartis website where you can make an inquiry U.S. Indication and Important Safety the air bubble through ophthalmic examination. Information Cataract Subretinal injection of LUXTURNA, LUXTURNA® (voretigene neparvovec-rzyl) for especially vitrectomy surgery, is associated with subretinal injection is an adeno-associated virus an increased incidence of cataract development vector-based gene therapy indicated for the and/or progression. treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Adverse Reactions Patients must have viable retinal cells as In clinical studies, ocular adverse reactions determined by the treating physicians. occurred in 66% of study participants (57% of injected eyes), and may have been related to Warnings and Precautions LUXTURNA, the subretinal injection procedure, the concomitant use of corticosteroids, or a Endophthalmitis may occur following any combination of these procedures and products. intraocular surgical procedure or injection. Use proper aseptic injection technique when The most common adverse reactions (incidence ≥ administering LUXTURNA, and monitor for and 5% of study participants) were conjunctival advise patients to report any signs or symptoms hyperemia (22%), cataract (20%), increased of infection or inflammation to permit early intraocular pressure (15%), retinal tear (10%), dellen treatment of any infection. (thinning of the corneal stroma) (7%), macular hole (7%), subretinal deposits (7%), eye inflammation Permanent decline in visual acuity may occur (5%), eye irritation (5%), eye pain (5%), and following subretinal injection of LUXTURNA. maculopathy (wrinkling on the surface of the Monitor patients for visual disturbances. macula) (5%). Retinal abnormalities may occur during or following the subretinal injection of LUXTURNA, Immunogenicity including macular holes, foveal thinning, loss of Immune reactions and extra-ocular exposure to foveal function, foveal dehiscence, and retinal LUXTURNA in clinical studies were mild. No hemorrhage. Monitor and manage these retinal clinically significant cytotoxic T-cell response to abnormalities appropriately. Do not administer either AAV2 or RPE65 has been observed. Study LUXTURNA in the immediate vicinity of the participants received systemic corticosteroids fovea. Retinal abnormalities may occur during or before and after subretinal injection of LUXTURNA following vitrectomy, including retinal tears, to each eye, which may have decreased the epiretinal membrane, or retinal detachment. potential immune reaction to either AAV2 or Monitor patients during and following the RPE65. injection to permit early treatment of these retinal abnormalities. Advise patients to report any signs or symptoms of retinal tears and/or Pediatric Use detachment without delay. Treatment with LUXTURNA is not recommended for patients younger than 12 months of age, Increased intraocular pressure may occur after because the retinal cells are still undergoing cell subretinal injection of LUXTURNA. Monitor and proliferation, and LUXTURNA would potentially be manage intraocular pressure appropriately. diluted or lost during the cell proliferation. The safety and ecacy of LUXTURNA have been Expansion of intraocular air bubbles Instruct established in pediatric patients. There were no patients to avoid air travel, travel to high significant dierences in safety between the elevations or scuba diving until the air bubble dierent age subgroups. formed following administration of LUXTURNA has completely dissipated from the eye. It may take one week or more following injection for the Please see the full U.S. Prescribing air bubble to dissipate. A change in altitude Information for LUXTURNA at while the air bubble is still present can result in sparktx.com/luxturna_us_prescribing_information. irreversible vision loss. Verify the dissipation of pdf. P-RPE65-US-790006 HIGHLIGHTS OF PRESCRIBING INFORMATION -------------------------------CONTRAINDICATIONS------------------------------ None. These highlights do not include all the information needed to use LUXTURNA safely and effectively. See full prescribing information for ------------------------WARNINGS AND PRECAUTIONS----------------------- LUXTURNA. • Endophthalmitis: Use proper aseptic injection technique and monitor for LUXTURNA (voretigene neparvovec-rzyl) intraocular suspension for signs and symptoms of infection. (5.1) subretinal injection • Permanent decline in visual acuity: Monitor for visual disturbances. Initial U.S. Approval: 2017 (5.2) • Retinal abnormalities: Monitor for macular abnormalities, retinal tears or -----------------------------INDICATIONS AND USAGE-------------------------- breaks. Do not inject in the immediate vicinity of the fovea. (5.3) LUXTURNA is an adeno-associated virus vector-based gene therapy • Increased intraocular pressure: Monitor and manage intraocular pressure indicated for the treatment of patients with confirmed biallelic RPE65 elevations. (5.4) mutation-associated retinal dystrophy. Patients must have viable retinal cells • Expansion of intraocular air bubbles: Air travel and/or scuba diving is as determined by the treating physician(s). (1) not recommended until any intraocular air bubbles have been absorbed. ------------------------DOSAGE AND ADMINISTRATION---------------------- (5.5) For subretinal injection only. • Cataract: Subretinal injection of LUXTURNA may result in cataract formation or increase in the rate of cataract progression. (5.6) • The recommended dose of LUXTURNA for each eye is 1.5 x 1011 -----------------ADVERSE REACTIONS---------- vector genomes (vg), administered by subretinal injection in a total volume of 0.3 mL. (2.1) The most common adverse reactions (incidence ≥ 5%) in the clinical trials • Perform subretinal administration of LUXTURNA to each eye on were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, separate days within a close interval, but no fewer than 6 days apart. (2.1) eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula). (6) • Recommend systemic oral corticosteroids equivalent to prednisone at 1 mg/kg/day (maximum of 40 mg/day) for a total of 7 days (starting 3 days before administration of LUXTURNA to each eye), and followed To report SUSPECTED ADVERSE REACTIONS, contact Spark by a tapering dose during the next 10 days. (2.1) Therapeutics, Inc. at 1-855-SPARKTX, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. ---------------------DOSAGE FORMS AND STRENGTHS---------------------- LUXTURNA is a suspension for subretinal injection, supplied in a 0.5 mL --------------------------USE IN SPECIFIC POPULATIONS--------------------- extractable volume in a single-dose 2 mL vial for a single administration in one Pediatric use: Use in infants under 12 months of age is not recommended 12 eye. The supplied concentration (5x10 vg/mL) requires a 1:10 dilution prior to because of potential dilution or loss of LUXTURNA after administration due administration. The Diluent is supplied in two single-use 2-mL vials. (3) to the active retinal cells proliferation occurring in this age group. (8.4) See 17 for PATIENT COUNSELING INFORMATION. FULL PRESCRIBING INFORMATION: CONTENTS* 8.2 Lactation 1 INDICATIONS AND USAGE 8.3 Females and Males of Reproductive Potential 2 DOSAGE
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