Issue:April 2018

GLOBAL REPORT – 2017 Global Drug Delivery & Formulation Report: Part 2, Notable Product Approvals of 2017


By: Kurt Sedo, VP of Operations, and Tugrul Kararli, PhD, President & Founder, PharmaCircle

Part 1: A Global Review

Part 2: Notable Product Approvals of 2017

Part 3: Notable Transactions and Technologies of 2017

Part 4: The Drug Delivery and Formulation Pipeline

 Introduction

What’s more interesting, the forest or the trees? Studying both can provide important information and insight. It’s the same with drug delivery and formulation products. In the first part of this series (March 2018 issue), we took a big picture look at 2017 approvals. In this second part, we look at the more interesting individual products approved last year. When looking at 2017 approvals as a whole, things don’t seem to be much different than 2016, or the previous years. The drug delivery and formulation forest, it seems, has stopped evolving. That has been the case for the better part of a decade.

But if you look closely, one finds a new “species,” gene/cell therapy products, among the expected injectable, oral, and inhalation product approvals. Whether these 2017 gene/cell therapy products are drug delivery or formulation enhanced is debatable. There is no question they will be. First-generation products always receive the attention of drug delivery and formulation to optimize therapeutic performance and create competitive advantage. The real question is whether this challenge will be taken up by the current generation of Drug Delivery and Formulation Professionals.

The Notable Product Approvals of 2017 are divided into three groups – Notable Drug Delivery& Formulation Approvals, Notable Drug-Device Approvals, and Other Notable Approvals. That last group includes the gene/cell therapy approvals. Individual approvals, if looked at closely, can provide important insight into the development, health, and future of the larger forest.

Notable Drug Delivery & Formulation Approvals of 2017

 Vantrela ER
Company: Teva (Cephalon)
Active(s): Hydrocodone Bitartrate
Molecule Type: Small Molecule
Indication: Pain Management
Delivery Route: Oral
Dosage Form: Oral Tablet
DD Category: Abuse Resistant Oral, Oral Barrier Film & Microparticles
Dosing: Twice Daily
First Approval: 2017-01-17 (USA)
Technology/Provider: OraGuard/Cima Labs (Teva)
Review Status: Standard, New Dosage Form – Discontinued (FDA)
Clinical Development/Approval Time: >7.2 Years
Notable: Approved yesterday and gone today. Vantrela ER was approved in January 2017, had its REMS proposal approved in May, and as of today is listed by the FDA as Discontinued. No reasons have been provided. With the ongoing concern about an opioid epidemic, it may be that companies are deciding that the commercial rewards do not outweigh the possible regulatory and public relations risks. Both Purdue Pharma (Hysingla ER) and Pernix (Zohydro ER) have previously introduced abuse-deterrent extended-release formulations of hydrocodone, so Vantrela ER would have been third to market with no obvious benefits.

Roxybond
Company: Daiichi Sankyo/Inspirion Delivery Sciences
Active(s): Oxycodone
Molecule Type: Small Molecule
Indication: Pain Management
Delivery Route: Oral
Dosage Form: Oral Tablet
DD Category: Abuse Resistant Oral, Oral Barrier Film & Microparticles
Dosing: Four to Six Times Daily
First Approval: 2017-04-20 (USA)
Technology/Provider: SentryBond/Inspirion Delivery Services
Review Status: Priority, New Formulation (FDA)
Clinical Development/Approval Time: >7.5 Years
Notable: Another abuse-deterrent formulation of oxycodone with a bit of a twist, an immediate-release presentation. Unlike Egalet’s Oxaydo, Roxybond does not incorporate aversive agents, relying on physical formulation properties to counter attempts to powder and insufflate or inject the product. There are no available forecasts for Roxybond. Egalet’s Oxaydo reported sales of $8 million in 2017.

Arymo ER
Company: Egalet
Active(s): Morphine Sulfate Pentahydrate
Molecule Type: Small Molecule
Indication: Chronic Pain
Delivery Route: Oral
Dosage Form: Oral Tablet
DD Category: Oral Erodible MR, Abuse Resistant, Oral
Dosing: Twice/Three Times Daily
First Approval: 2017-01-19 (USA)
Technology/Provider: Guardian / Egalet
Review Status: Standard, New Formulation (FDA)
Clinical Development/Approval Time: >3.8 Years
Notable: Arymo ER is another product in the increasingly long list of recent approvals for abuse-deterrent versions of well-characterized opioids, in this case morphine. It’s not clear how physicians and formularies will embrace an abuse-deterrent formulation of a product that is not generally considered a first choice for abuse in the face of generic competitors. Arymo ER uses a physical approach to abuse deterrence that eliminates the issues seen with other morphine abuse-deterrent products that incorporate an opioid antagonist. Analysts estimate Arymo ER potential sales of $100 million by 2022.

Rebinyn

Company: Novo Nordisk
Active(s): Nonacog Beta Pegol
Molecule Type: Protein
Indication: Hemophilia B
Delivery Route: Injection
Dosage Form: Lyophilized Powder for Solution
DD Category: Prodrugs PEG Polymer, Reconstitution Systems
Dosing: Once Weekly
First Approval: 2017-05-31 (USA)
Technology/Provider: GlycoPEGylation Technology/Novo Nordisk, MixPro/Novo Nordisk
Review Status: Standard, BLA (FDA)
Clinical Development/Approval Time: >7.7 Years
Notable: The pipeline of innovative PEGylated products has shrunk over the past 2 decades as many opportunities have been previously captured or eliminated by virtue of longer half-life actives. Rebinyn is a 40 kD PEGylated version of recombinant Factor IX. In terms of presentation, it is only available as a lyophilized single-dose vial for reconstitution. The product is approved for acute use only. Analysts estimate global sales on the order of $150 million by 2023.

Cotempla XR-ODT
Company: Neos Therapeutics
Active(s): Methylphenidate
Molecule Type: Small Molecule
Indication: ADHD
Delivery Route: Oral
Dosage Form: Oral Tablet
DD Category: Conventional Melt Tablets, Taste Masking, Oral Ion Exchange MR
Dosing: Once Daily
First Approval: 2017-06-19 (USA)
Technology/Provider: Neos RDIM/Neos Therapeutics
Review Status: Standard, New Dosage Form (FDA)
Clinical Development/Approval Time: >4.2 Years
Notable: The ADHD market has largely escaped the attention of regulators and the public in terms of potential abuse liability. This has encouraged companies to develop differentiated product formulations. Cotempla XR-ODT is a 12-hour extended-release melt tablet formulation of methylphenidate. There is no evidence that Neos is developing a liquid methylphenidate formulation comparable to their Adzenys ER. Analysts forecast annual sales reaching $80 million by 2022.

Adzenys ER
Company: Neos Therapeutics
Active(s): Levamphetamine, Dextroamphetamine
Molecule Type: Small Molecule
Indication: ADHD
Delivery Route: Oral
Dosage Form: Oral Suspension
DD Category: Oral Liquid MR, Oral Ion Exchange MR, Taste Masking
Dosing: Once Daily
First Approval: 2017-09-15 (USA)
Technology/Provider: Neos DTRS/Neos Therapeutics
Review Status: Standard, New Dosage Form (FDA)
Clinical Development/Approval Time: >4 Years
Notable: The ADHD market has largely escaped the attention of regulators and the public in terms of potential abuse liability, leading companies to develop differentiated product formulations. Adzenys ER is the third Neos approval for ADHD. This once-daily liquid extended-release amphetamine formulation uses the same technology as Neos’ Adzenys XR-ODT amphetamine product, in a liquid formulation. Analysts are suggesting the product will have modest annual sales that reach $30 million by 2022.

Mavenclad
Company: Merck Serono Europe
Active(s): Cladribine
Molecule Type: Small Molecule
Indication: Multiple Sclerosis, Relapsing Remitting
Delivery Route: Oral
Dosage Form: Oral Tablet
DD Category: Cyclodextrins/Solubilizers
Dosing: Single Dose Daily According to Protocol
First Approval: 2017-08-25 (EU)
Technology/Provider: Ivax/Teva Cyclodextrin-Complexes/Teva Pharmaceutical Industries
Review Status: Standard (EMA)
Clinical Development/Approval Time: >12.3 Years
Notable: Approved as far back as 1993 as an injectable for the treatment of a variety of hematological malignancies, cladribine has been reformulated as an oral treatment for highly relapsing multiple sclerosis. Dosing is unique with a recommended dose of 3.5 mg/kg (245 mg/70kg) over a 2-year period. An initial filing in 1997 by Ortho-Clinical was rejected. Some 20 years later, it is now approved in Europe, Australia, and Canada. The product is in Phase 3 development in the USA, after a 2011 rejection by the FDA. Sales are expected to grow to $200 million annually by 2022.

Mydayis
Company: Shire/Supernus
Active(s): Mixed Amphetamines
Molecule Type: Small Molecule
Indication: ADHD
Delivery Route: Oral
Dosage Form: Oral Capsule
DD Category: Oral Barrier Film & Microparticles, Oral Enteric/Delayed Release
Dosing: Once Daily
First Approval: 2017-06-20 (USA)
Technology/Provider: None
Review Status: Standard, New Dosage Form (FDA)
Clinical Development/Approval Time: >13.3 Years
Notable: The ADHD market has largely escaped the attention of regulators and the public in terms of potential abuse liability. This has led companies to develop differentiated product formulations. Shire, with its substantial ADHD market presence announced approval of Mydayis, another once-daily amphetamine product approval in June of last year. The product is distinguished by a longer duration of action, up to 16 hours. Analysts are relatively bullish on the product, estimating sales exceeding $400 million annually by 2022.

Zilretta
Company: Flexion Therapeutics
Active(s): Triamcinolone Acetonide
Molecule Type: Small Molecule
Indication: Osteoarthritis
Delivery Route: Injection
Dosage Form: Injection Powder for Suspension
DD Category: Biodegradable PLGA Microcaps/Implants
Dosing: Single Dose
First Approval: 2017-10-06 (USA)
Technology/Provider: Flexion PLGA Microspheres/Flexion Therapeutics
Review Status: Standard, New Formulation (FDA)
Clinical Development/Approval Time: >5.3 Years
Notable: Zilretta represents a logical therapeutic option for a common use of corticosteroids; intra-articular injection of the knee for the treatment of osteoarthritis. In this case, triamcinolone is formulated with biodegradable PLGA to provide a 4-fold extension of half-life. This appears to be the first long-acting steroid to be approved for this indication. Analysts are estimating annual sales reaching almost $500 million annually by 2022. Sometimes simple solutions to common problems can be profitable.

Lyrica CR Tablets
Company: Pfizer
Active(s): Pregabalin
Molecule Type: Small Molecule
Indication: Neuropathic Pain, Post-Herpetic Neuralgia
Delivery Route: Oral
Dosage Form: Oral Tablet
DD Category: Gastro Retentive, Oral Matrix MR
Dosing: Once Daily
First Approval: 2017-10-11 (USA)
Technology/Provider: Pfizer/Internal
Review Status: Standard, New Formulation (FDA)
Clinical Development/Approval Time: >7.0 Years
Notable: Pfizer has extended their significant Lyrica franchise with the approval of a once-daily formulation of pregabalin to combat impending generics. The product uses gastroretentive and oral matrix modified-release technologies to move from twice daily to once daily. 2017 also saw the approval of Lyrica OD in Japan, an ODT twice-daily formulation of pregabalin to improve compliance for elderly patients with fibromyalgia.

Sublocade
Company: Indivior
Active(s): Buprenorphine
Molecule Type: Small Molecule
Indication: Opioid Dependence
Delivery Route: Injection
Dosage Form: Injection Solution
DD Category: Biodegradable Gel/Suspension
Dosing: Up to 6 Months
First Approval: 2017-11-30 (USA)
Technology/Provider: ATRIGEL/Tolmar, Novelion Therapeutics, RB Group
Review Status: Priority, Orphan, New Dosage Form (FDA)
Clinical Development/Approval Time: >7.1 Years
Notable: Faced with branded and generic competitors to their sublingual buprenorphine opioid dependence franchise, Indivior has added a long-acting depot formulation of buprenorphine for the same indication. It’s not clear how physicians and patients will respond to this treatment option. Analysts have Sublocade annual sales reaching $800 by 2023, but Titan’s 6-month Probuphine buprenorphine implant is apparently experiencing significant commercial and regulatory challenges. Perhaps an injection rather than an implant will make all the difference.

Oncaspar
Company: Baxalta (Shire)
Active(s): Asparaginase
Molecule Type: Protein
Indication: Acute Lymphocytic Leukemia
Delivery Route: Injection
Dosage Form: Lyophilized Powder for Solution
DD Category: Prodrugs PEG Polymer
Dosing: Once every 2 weeks
First Approval: 2017-12-13 (EU)
Technology/Provider: 1st Generation PEGylation / Enzon
Review Status: Standard (EMA)
Clinical Development/Approval Time: Unknown
Notable: Oncaspar has been a mainstay of pediatric ALL treatment for almost 3 decades. During much of that period, there have been periodic supply issues related to the availability of the asparaginase enzyme, and product stability issues. The development of a more stable formulation will go a long way to improving access. In the case of a hospital-administered product, the extra step of reconstitution is a small inconvenience for a secured supply of this life-saving drug. Analysts expect annual sales of Oncaspar to grow to more than $250 million by 2022.

Notable Drug-Device Approvals of 2017

Instanyl Doseguard
Company: Takeda Pharma, Nycomed
Active(s): Fentanyl Citrate
Molecule Type: Small Molecule
Indication: Breakthrough Pain
Delivery Route: Nasal
Dosage Form: Nasal Spray Metered
DD Category: Nasal Spray Pumps/Devices, Drug Delivery Compliance
Dosing: No less than 2 to 4 Hours
First Approval: 2017-05-15 (EU)
Technology/Provider: Aptar Nasal/SL Metered Dose eDevice/Aptar Pharma
Review Status: Standard (EMA)
Clinical Development/Approval Time: Unknown
Notable: Instanyl Doseguard represents a compliance enhancement to Instanyl, a nasal fentanyl formulation first approved in Europe in 2009. The device features a large and highly visible dose-counting display (number of doses left) and flashing display. The electronic display shows whether the nasal spray is locked or ready for use, and the “e-Lockout” feature counts and displays the number of actuations, preventing device actuation for a period of time after a pre-defined number of actuations.

Enbrel Mini Cartridge
Company: Amgen
Active(s): Etanercept
Molecule Type: Protein (Fusion)
Indication: Ankylosing Spondylitis, Juvenile Arthritis, Psoriasis, Psoriatic Arthritis, Rheumatoid Arthritis
Delivery Route: Injection
Dosage Form: Injection Solution
DD Category: Prodrugs Antibody- ADC, Autoinjectors, Reusable
Dosing: Once/Twice Weekly
First Approval: 2017-09-14 (USA)
Technology/Provider: AutoTouch/Amgen
Review Status: Supplement (FDA)
Clinical Development/Approval Time: Unknown
Notable: The Enbrel Mini Cartridge system has been designed as a follow on to the Prefilled Syringes and Pen presentations. The Enbrel Mini Cartridge is a single- use presentation that can only be used with the AutoTouch injector. The AutoTouch injector is intended to ensure more consistent dosing by limiting the number of potential points of patient confusion. AutoTouch and the Mini Cartridge system may be used as a platform for other Amgen products, including Neupogen, Nplate, and Aranesp.

Trelegy Ellipta Inhalation Powder
Company: Glaxo SmithKline
Active(s): Fluticasone Furoate, Umeclidinium Bromide, Vilanterol Trifenatate
Molecule Type: Small Molecules
Indication: Chronic Obstructive Pulmonary Disease
Delivery Route: Inhalation
Dosage Form: Inhalation Powder
DD Category: DPI-Dry Powder Inhalers, Combination/Incompatible
Dosing: Once Daily
First Approval: 2017-09-18 (USA)
Technology/Provider: Ellipta/Glaxo SmithKline
Review Status: Standard, New Formulation (FDA)
Clinical Development/Approval Time: >5.4 Years
Notable: Trelegy Ellipta represents a trifecta of sorts, providing COPD patients with a three-component therapy in a single inhalation, once a day. It’s notable that the FDA approved the product as a New Formulation (Type 5) rather than either a New Dosage Form (Type 3) and/or New Combination (Type 4), suggesting that the approval was largely based on the previous approval of the separate two-component Ellipta products. Analysts expect sales to ramp up slowly, hitting $1 billion annually by 2022.

XHANCE

Company: OptiNose US
Active(s): Fluticasone Propionate
Molecule Type: Small molecule
Indication: Nasal Polyposis
Delivery Route: Nasal
Dosage Form: Nasal Spray Metered, Suspension
DD Category: Nasal Spray Pumps/Devices
Dosing: Once/Twice Daily
First Approval: 2017-09-18 (USA)
Technology/Provider: OptiNose Liquid Delivery Device/OptiNose US
Review Status: Standard, New Formulation (FDA)
Clinical Development/Approval Time: >5.2 Years
Notable: XHANCE is OptiNose’s nasal polyposis product follow up to 2016’s ONZETRA Xsail. XHANCE nasal spray incorporates the same unique delivery device that uses the patient’s breath to power the nasal inhalation while closing the soft palate to limit dispersion of the dose. Restricting fluticasone to the sinus cavity is critical when dosing at 4-times the levels commonly prescribed with nasal steroids used for the treatment of seasonal allergies.

Bydureon BCise
Company: AstraZeneca
Active(s): Exenatide
Molecule Type: Peptide
Indication: Diabetes, Type 2 Delivery Route: Injection
Dosage Form: Injection Solution
DD Category: Biodegradable PLGA Microcaps/Implants, Disposable Autoinjectors, Single Use
Dosing: Once Weekly
First Approval: 2017-10-20 (USA)
Technology/Provider: BCise/AstraZeneca
Review Status: Standard, Type-3
Clinical Development/Approval Time: >7.0 Years
Notable: Once-weekly dosing is not a competitive advantage if it requires the use of a clunky dual-chamber autoinjector. The new BCise autoinjector provides significant patient-use improvements over the older dual- chamber LyoTwist Trio device. Dose preparation time is reduced significantly, and the new BCise autoinjector better disguises the needle injection process. It increasingly seems that competition in the Type 2 diabetes market has become an “arms race” of ever improved formulations and injection devices. Bydureon BCise is expected to help grow the overall Bydureon franchise from $577 million in 2017 to almost $900 million by 2022.

Ozempic
Company: Novo Nordisk
Active(s): Semaglutide
Molecule Type: Peptide
Indication: Diabetes, Type 2
Delivery Route: Injection
Dosage Form: Injection Solution
DD Category: Injection Pens, Disposable
Dosing: Once/Twice Weekly
First Approval: 2017-12-05 (USA)
Technology/Provider: Flex Touch/Novo Nordisk
Review Status: Standard, New Molecular Entity (FDA)
Development/Approval Time: >10.5 Years
Notable: After a period of new outpatient injectables being introduced with a logical progression of vial/syringe presentations, followed by prefilled syringes, and then pen devices, products are increasingly being introduced with only an injection pen option. And dosing has moved from several times daily to once daily to once or twice weekly, significantly simplifying the treatment of Type 2 diabetes. Ozempic, it seems, will be major competition for Lilly’s Trulicity. The real opportunity for semaglutide may be provided by an oral dosage formulation in Phase 3 trials. Ozempic annual sales are forecast to exceed $2.5 billion by 2022.

Abilify MyCite
Company: Otsuka Pharmaceutical
Active(s): Aripiprazole
Molecule Type: Small Molecule
Indication: Bipolar Disorder, Depression, Schizophrenia
Delivery Route: Oral
Dosage Form: Oral Tablet
DD Category: Drug Delivery Compliance, Ingestible Delivery Devices
Dosing: Once Daily
First Approval: 2017-11-13 (USA)
Technology/Provider: Proteus Ingestion Event Marker/Proteus Digital Health
Review Status: Standard, New Combination (FDA)
Development/Approval Time: >7.5 Years
Notable: Abilify MyCite combines the well-validated CNS agent aripiprazole with an ingestible sensor in an oral tablet presentation. The sensor tracks ingestion of the tablet by means of an external wearable patch that communicates the information to a smartphone application. The full potential for this type of medication-sensor combination is yet to be fully realized, but at this point, the major objective seems to track compliance, an issue with some psychiatric conditions. Sales are likely to be negligible, and will largely act as a test case for more commercially attractive opportunities.

Nyxoid
Company: Mundipharma International
Active(s): Naloxone Hydrochloride
Molecule Type: Small Molecule
Indication: Substance Abuse
Delivery Route: Nasal
Dosage Form: Nasal Spray
DD Category: Nasal Formulations
Dosing: Single Dose
First Approval: 2017-11-10 (EU)
Technology/Provider: None
Review Status: Standard (EMA)
Development/Approval Time: Unknown
Notable: Mundipharma’s nasal naloxone was approved in Europe on the basis of a clinical literature review and a 5-part open label, randomized, single-dose, crossover study in healthy volunteers. The primary trial endpoints related to the Nyxoid’s pharmacokinetic properties. In summary, a very limited investment to improve opioid overdose outcomes.

Other Notable Approvals of 2017

Vyxeos
Company: Celator Pharmaceuticals (Jazz Pharmaceuticals)
Active(s): Cytarabine, Daunorubicin
Molecule Type: Small Molecule
Indication: Acute Myelogenous Leukemia
Delivery Route: Injection
Dosage Form: Injection Lyophilized Powder for Suspension
DD Category: NP liposome, Combination/Incompatible
Dosing: Per Protocol
First Approval: 2017-08-03 (USA)
Technology/Provider: CombiPlex Drug-Ratio Technology/Celator Pharmaceuticals
Review Status: Priority, Orphan, New Combination (FDA)
Clinical Development/Approval Time: >15.9 Years
Notable: Vyxeos uses Celator’s Combiflex Drug-Ratio technology that identifies molar ratios of drugs that deliver a synergistic benefit and locks the desired ratio in a “nano-scale” liposomal drug delivery vehicle that maintains the ratio in patients. Using two well-characterized actives, cytarabine and daunorubicin, Vyxeos received Priority and Orphan designation from the FDA as a first-line treatment for AML, a very competitive indication. Analysts have forecast Vyxeos annual sales of more than $300 million by 2022.

Gene Cell Therapy

Kymriah
Company: Novartis/Univ. of Pennsylvania
Active(s): Tisagenlecleucel
Molecule Type: T-Cell
Indication:m Acute Lymphocytic Leukemia
Delivery Route: Injection
Dosage Form: Injection Suspension
DD Category: Retroviral Transfection, Cell Expansion
Dosing: Once Only
First Approval: 2017-08-30 (USA)
Technology/Provider: CART-19/Univ. of Pennsylvania
Review Status: Priority, BLA (FDA)
Clinical Development/Approval Time: >7.5 Years
Notable: CAR-T (Chimeric Antigen Receptor, T-cell) immunotherapies are the great hope for recruiting a patient’s immune system to selectively recognize and attack cancer cells. At this stage, Kymriah is limited to patients up to the age of 25 with relapsed or refractory Acute Lymphoblastic Leukemia. Additional indications are in clinical development. Analysts forecast sales of more than $1 billion annually by 2022, mostly in Europe.

Yescarta
Company: Kite Pharma (Gilead Sciences)
Active(s): Axicabtagene Ciloleucel
Molecule Type: T-Cell
Indication: B-Cell Lymphomas, DLBCL, Follicular Lymphoma, NHL
Delivery Route: Injection
Dosage Form: Injection Suspension
DD Category: Retroviral Transfection, Cell Expansion
Dosing: Once Only
First Approval: 2017-10-18 (USA)
Technology/Provider: CAR/National Cancer Institiute
Review Status: Priority, Orphan, Breakthrough Therapy, BLA (FDA)
Clinical Development/Approval Time: >8.8 Years
Notable: CAR-T (Chimeric Antigen Receptor, T-cell) involves the tedious and costly process of transfecting the patient’s T-cells ex vivo with a retroviral construct coding the specific CD-19 targeted antigen, expanding the cells and reinfusing them to the patient. At this stage, treatment is limited to relapsed or refractory patients with B-Cell Lymphomas, DLBCL, Follicular Lymphoma, or NHL. Analysts forecast Yescarta sales of more than $2 billion annually by 2022.

Luxturna Injection
Company: Spark Therapeutics
Active(s): Voretigene Neparvovec
Molecule Type: AAV Gene Vector
Indication: Leber’s Congenital Amaurosis, Retinitis Pigmentosa
Delivery Route: Surgical Insertion
Dosage Form: Injection Suspension
DD Category: Adeno-Associated Virus Vectors
Dosing: Once Only
First Approval: 2017-12-19 (USA)
Technology/Provider: Spark AAV Vectors/Children’s Hospital of Philadelphia, Spark Therapeutics
Review Status: Priority, Orphan, Rare Pediatric, BLA
Clinical Development/Approval Time: >10.9 Years
Notable: This seems to be an ideal indication for early gene therapy to explore: a clear mechanism of action, a localized condition not amenable to traditional therapies, and one-time administration. It makes sense in terms of cause and effect, the need is high without options, and the potential for a life-threatening adverse event is limited. The expanded safety information that will be provided by commercial use of Luxturna may validate the use of the Spark AAV vector system for less localized applications. Despite the very limited indication, analysts have forecast annual sales of more than $500 million by 2022.

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