The FDA recently approved a new therapy, Litfulo (ritlecitinib) from Pfizer, Inc., a once-daily oral treatment, for individuals 12 years of age and older with severe alopecia areata.

Alopecia areata is an autoimmune disease characterized by patchy or complete hair loss on the scalp, face, or body.  It has an underlying immuno-inflammatory pathogenesis and develops when the immune system attacks the body’s hair follicles, causing hair to fall out.  This hair loss often occurs on the scalp, but it can also affect eyebrows, eyelashes, facial hair, and other areas of the body.  Alopecia totalis (total scalp hair loss) and alopecia universalis (total body hair loss) are types of alopecia areata.

Alopecia Areata impacts nearly 7 million people in the U.S. and approximately 147 million people globally and can affect people of any age, gender, race, or ethnicity and can cause considerable burden beyond hair loss.  Nearly 20% of people with alopecia areata are diagnosed before the age of 18.

The therapy was approved with a Boxed Warning that includes Serious Infections, Mortality, Malignancy, Major Adverse Cardiovascular Events (Mace), and Thrombosis.

Pfizer announced that a full year supply of Litfulo carries a list price of $49,000, similar to other specialty dermatologic treatments.

Distribution and logistics for the therapy were not announced but it is highly likely it will launch through specialty pharmacy limited distribution.

CLICK HERE to access prescribing information

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FDA Approves Pfizer’s LITFULO for Adults and Adolescents with Severe Alopecia Areata

LITFULO is the first and only treatment for severe alopecia areata approved for patients 

June 23, 2023 — NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved LITFULO (ritlecitinib), a once-daily oral treatment, for individuals 12 years of age and older with severe alopecia areata. The approved recommended dose for LITFULO is 50 mg. It is the first and only treatment approved by the FDA for adolescents (12+) with severe alopecia areata.

“While patients may start to develop symptoms of alopecia areata at any age, most people start showing signs in their teens, twenties, or thirties,” said Dr. Brittany Craiglow, Associate Professor Adjunct – Dermatology at Yale School of Medicine. “LITFULO is a particularly important treatment option for younger patients with substantial hair loss, who often struggle with such a visible disease.”

LITFULO is a kinase inhibitor which inhibits Janus kinase 3 (JAK3) and the tyrosine kinase expressed in hepatocellular carcinoma (TEC) family of kinases.

“LITFULO is an important treatment advancement for alopecia areata, an autoimmune disease that previously had no FDA-approved options for adolescents and limited options available for adults,” said Angela Hwang, Chief Commercial Officer, President, Global Biopharmaceuticals Business, Pfizer. “With today’s approval, adolescents and adults who struggle with substantial hair loss have an opportunity to achieve significant scalp hair regrowth.”

The FDA approval was based on results of clinical trials in alopecia areata. The ALLEGRO Phase 2b/3 trial, which enrolled 718 patients with 50% or more scalp hair loss as measured by the Severity of Alopecia Tool (SALT), evaluated the efficacy and safety of LITFULO at 118 sites in 18 countries. In this pivotal study, 23% of patients treated with LITFULO 50 mg had 80% or more scalp hair coverage (SALT≤20) after six months compared to 1.6% with placebo. The efficacy and safety of LITFULO were consistent between adolescents (12 through 17 years of age) and adults (18 years of age and older). The most common adverse events (AEs) reported in at least 4% of patients with LITFULO include headache (10.8%), diarrhea (10%), acne (6.2%), rash (5.4%), and urticaria (4.6%). Full results from the ALLEGRO Phase 2b/3 study were published by The Lancet in April 2023.

“People living with alopecia areata are often misunderstood, and their experience is frequently trivialized as ‘just hair.’ However, it is a serious autoimmune disease that can have considerable negative impact beyond the physical symptoms,” said Nicole Friedland, President and Chief Executive Officer of the National Alopecia Areata Foundation (NAAF). “We believe the approval of LITFULO is a significant advancement for the treatment of alopecia areata, particularly for teens. It’s exciting to see more FDA-approved treatments becoming available for this community.”

View the full Prescribing Information. If it is not currently available via this link, it will be visible as soon as possible as we work to finalize the document. Please check back for the full information shortly.

Every day we scan hundreds of articles and press releases to find items, events, trends, developments and other flotsam and jetsam related to specialty pharmacy, specialty infusion and the emerging pharmaceutical marketplace in general. Today we ran across an article published in the Irish ECHO that has some interest for us here in this dear land across the Irish Sea.

A company called HealthSolutions (yep, no space) is making some inroads with US-based specialty pharmacies, including Accredo, with a spiffy new device for patients on injectable therapy. Think of it as AI meets sharps container. It is called the SmartSharpsBin….. catchy name huh?. Now a sharps container by itself is not exactly hot news but one that communicates with its owner to remind them of their next scheduled injection and will alert them if the box isn’t fed the last now used scheduled syringe is noteworthy. And, yes, there is also an app for that on your phone! When the container is about to be full, a new container is automatically sent to the patient who then uses that same mailing container to return the used sharps container….. neat, HUH?

Supposedly, health insurers are reimbursing for the device. And, anyone can obtain one independently via membership (multiple plans are available starting at 12 months for a flat $420 or $35 monthly.)

Whether the concept takes hold remains to be seen…. especially at that price point. None the less, this gizmo is one of the newest tech offerings to promote patient compliance that we’ve seen in some time and, who knows, it may be the start of a new thang.

CLICK HERE to visit the SmartSharpsBin web site.

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HealthBeacon Share Price Jumps by 111% in the Past Month

— HealthBeacon has seen its share price jump by 111% in the past month.

— The Inchicore company, which listed on Euronext Dublin in 2021, develops smart tools to assist patients to manage their medications from their homes.

HealthBeacon’s IPO raised €25m, with the company reporting a market capitalisation of €98.4m on the day of its public listing. This figure now stands at around €32m on Euronext Dublin, while shares have slid around 58 per cent in the past year.

Jim Joyce, the company’s founder and chief executive attributes this to a surge in activity in the US, with a few key areas giving “all that optimism that we’ve got the right product fit”. “We’ve done a number of deals with specialty pharmacy organisations in the US,” he said to the Independent. “Organisations like Accredo, where they integrated the technology into their fundamental offering. “That means if you’re on a high-value injectable medication and you’re working with certain large pharmacy systems, you’re going to be offered the HealthBeacon device as part of going on that medication.

More US partnerships are coming down the line with speciality pharmacy organisations, pharma companies, as well as clinical practices, Mr Joyce says.  HealthBeacon’s offerings are reimbursed by insurers in the US.

HealthBeacon has over 20 employees in the US market, with offices in Boston and Orlando. Overall, the company’s headcount stood at around 67 at the end of last year.

The company’s client base grew to 30 last year from 23 in 2021. Sales rose slightly to €2.25m from €2.2m the prior year, while its losses pretax grew to €13m. This was attributed to a global chip shortage and energy crisis, which also weighed on gross margins. However, Joyce is upbeat about what the future holds as the business predicts annualised revenue of €25m by the end of March next year.

Are you familiar with the Preserving Patient Access to Home Infusion Act (PPAHIA)?

You probably should be.  It has been kicking around the halls of Congress since 2021…. without passage.  However, like Whack-a-Mole, it keeps popping up in each new Congress because it has popular appeal and even bipartisan support, at least in the Senate.

If it ever gets passed, the bill will clarify that drugs shall be reimbursement and at what rates.  No small achievement for the home health industry. There’s a big  sticking point however ….. hospitals. The more home infusions the fewer hospital outpatient infusions and related hospital revenues….. and the hospital lobby is on guard. As we know, hospital outpatient infusions generally cost multiples of the same service delivered in the home. 

Besides being able to save millions for patients and Medicare/Medicaid, the bill seeks to correct some changes that were included in the 2018 Cures and Bipartisan Budget Acts to create a professional services benefit for Medicare Part B home infusion drugs. That tinkering “resulted in CMS improperly implementing the benefit by requiring a nurse to be physically present in the patient’s home in order for providers to be reimbursed. As a result, provider participation in Medicare’s home infusion benefit has dropped sharply and beneficiaries have experienced reduced access to home infusion over the last several years.”

Since little legislation seems to be leaking out of Washington these days this bill may have to wait another year…. or more…. before it ever gets passage as common sense might dictate. 

Oh well…

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Lawmakers Reintroduce Bipartisan, Bicameral Legislation to Increase Access to Medicare Home Infusion Benefit

Jun 14 2023 — WASHINGTON – U.S. Sens. Mark R. Warner (D-VA) and Tim Scott (R-SC) joined by U.S. Reps. Vern Buchanan (R-FL-16), Chairman of the House Ways & Means Health Subcommittee, Debbie Dingell (D-MI-06), Diana Harshbarger (R-TN-01), and Terri Sewell (D-AL-07) today reintroduced legislation that will ensure patients maintain access to home infusion therapy. The Preserving Patient Access to Home Infusion Act protects access to Medicare’s home infusion benefit by making clear that pharmacy services for home infusion therapy can be reimbursed and sets an appropriate rate for such services.

“We have seen for years that patients are better off when they can receive quality care from the comfort of their own homes,” said Sen. Warner.   “This legislation would ensure that……..

…………..press release continues

CLICK HERE to read the full press release

FDA has now approved four gene therapies within the past ninety days.

That’s got to be a record!

The FDA recently approved a new infused gene therapy, Elevidys (delandistrogene moxeparvovec-rokl) from Sarepta Therapeutics.   Elevidys is administered as a single-dose intravenous infusion and is the first and only gene therapy approved for Duchenne. It is an adeno-associated virus-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.  Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Elevidys addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin. This accelerated approval is based on an increase in Elevidys micro-dystrophin protein expression in skeletal muscle.

Elevidys  is the sixth approved therapy in Duchenne and the first therapy to replace the missing dystrophin protein by using a modified smaller version of the dystrophin gene, called micro-dystrophin, to produce a modified micro-dystrophin protein .Duchenne is a fatal genetic disorder that slowly robs patients of their muscle strength.

The therapy was approved without a black box warning but alerts were cited for Acute Serious Liver Injury, Immune-mediated Myositis, and Myocarditis.

Sarepta announced that Elevidys will launch at a one-time treatment cost of $3.2 million.

Details related to distribution and logistics were not released at time of approval, but industry sources have confirmed that at least one specialty pharmacy will be selected as the company’s specialty distribution partner. 

CLICK HERE for full prescribing information

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Sarepta Therapeutics Announces FDA Approval of Elevidys, the First Gene Therapy to Treat Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jun. 22, 2023– Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug Administration (FDA) accelerated approval of Elevidys (delandistrogene moxeparvovec-rokl), …………

CLICK HERE to access the full Sarepta press release

Not all specialty pharmacies operate a compounding line of business….. but there are a number of SPs that do. If your pharmacy is into compounding, then you should already be aware of the new standards that are going into effect on November 1^st. If your specialty pharmacy isn’t into compounding, then read on regardless….. and learn something new about your industry.

The new standards are being published by the USP which stands for United States Pharmacopeia, an industry “bible” published annually by this now over 200-year old nonprofit organization. USP is not a regulatory body or an accrediting agency . Rather, USP is an independent, scientific nonprofit organization focused on building trust in the supply of safe, quality medicine and works to strengthen the global supply chain. USP healthcare standards and solutions are practical tools that healthcare practitioners use to help ensure consistency in operations, foster timely decision making, and quality of patient care. 

As noted in the article, USP standards regularly filter down into regulations that might be federal or state, and/or incorporated by accrediting organizations. As such, it behooves any compounding pharmacy, to get into compliance before an inspection or a reaccreditation. Take special note of the substantive changes to Chapters 787 and 800.

Visit the USP website to learn more.

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USP Countdown: Where Are You With Less Than Five Months to Go?

JUNE 21, 2023

With less than five months left before the revised USP compounding-related chapters <795>, <797>, <800> and <825> become compendially applicable on Nov. 1, 2023, experts at the ASHP 2023 Summer Meetings and Exhibition urged hospital pharmacy leaders to complete a gap analysis if they have not done so already, be aware of additional and possibly more stringent requirements that their states may have in place over and above the USP minimum standards, and use the available survey tool from The Joint Commission (if they’re accredited by the organization). 

CLICK HERE to read the full article

Another sonic boom could be heard (well….. at least by dogs) recently when the FDA approved yet another gene therapy! This time the news was truly noteworthy, as the therapy in question has been the aspirational hope of a well known, rare disease population for neigh on three decades. The approved therapy is Roctavian (valoctocogene roxaparvovec-rvox) from BioMarin Pharmaceutical, a gene therapy for the treatment of adults with severe hemophilia-A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.

Roctavian  is a one-time, single-dose infusion and  is the first approved gene therapy for severe hemophilia A in the U.S. 

BioMarin said it estimates only about 2,500 US patients would be eligible for the therapy.  Patients with certain underlying health conditions and problems are excluded from the prescribing information.  The company went on to explain that Roctavian was approved only for patients with severe disease who test negative for an antibody that attacks the virus Roctavian uses to deliver its helpful genetic material into cells. Upwards of 20,000 males in the US have hemophilia-A meaning that about 17,000 people are not candidates for this particular one-and-done therapy. However, this approval only fuels the hopes of the remaining hemophilia-A candidates.

CLICK HERE to access prescribing information

BioMarin claims that Roctavian will come to market at $2.9 million….. which is arguably not expensive when annual costs for traditional blood factors can run as much as $800,000 annually for a typical patient. They also pointed out that the price is less than the $3.5 million for a similar gene therapy for hemophilia-B approved last fall.

BioMarin did not announce details for logistics and distribution.

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BioMarin finally secures FDA approval of hemophilia gene therapy

After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.

June 29, 2023 — The Food and Drug Administration approved on Thursday the first gene therapy for the most common form of hemophilia, clearing the way for what patients, doctors and the medicine’s developer hope could be a one-time treatment for the rare bleeding disorder.

BioMarin Pharmaceutical, the California-based company behind the therapy, plans to sell it under the brand name Roctavian. It’s specifically meant to treat hemophilia A, which is caused by genetic mutations that inhibit the production of a key blood-clotting protein known as Factor VIII.

The company has set a list price of $2.9 million in the U.S., higher than the therapy’s cost in Europe, where it’s priced at about………………article continues

CLICK HERE to access the full article

The FDA recently approved a new infused therapy, Columvi (glofitamab-gxbm) from Genentech, for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) not otherwise specified or large B-cell lymphoma (LBCL) arising from follicular lymphoma, after two or more lines of systemic therapy. Columvi is a T-cell engaging bispecific antibody in non-Hodgkin’s lymphoma, joining recently approved Lunsumio indicated for follicular lymphoma.

DLBCL is considered hard to treat. It is the most common form of non-Hodgkin’s lymphoma. People with DLBCL who relapse and are refractory  progress rapidly and have not been well served by prior therapies. The company pointed out that Columvi is “….an off-the-shelf, fixed-duration treatment providing durable response rates, we believe Columvi could change the way this aggressive lymphoma is treated.” “Further, Columvi offers “a chance for complete remission with a fixed-duration immunotherapy and that such remissions can potentially be sustained after the end of their treatment.”

The fixed-duration for Columvi, as detailed in the package insert, is 12 cycles at 21 day intervals.

Columvi was approved with a black box warning for Cytokine Release Syndrome. 

The company said that the expected cost for Columvi will be about $350,000 for the full course and would be available in the United States in the coming weeks.

Distribution details  were not disclosed. Given its cost and black box warning it is likely that product access will include specialty pharmacy distribution.  

CLICK HERE to access prescribing information

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FDA Approves Genentech’s Columvi for People with Relapsed or Refractory Diffuse Large B-Cell Lymphoma

Pivotal study showed durable responses, with a 56% overall response rate, a 43% complete response (remission) rate, and a median duration of response of 1.5 years (18.4 months)

Given over a fixed period of time, Columvi provides patients with a treatment end date and potential time off treatment.

South San Francisco, CA — June 15, 2023 –Genentech, a member of the Roche Group, announced today that the U.S. Food and Drug Administration (FDA) has approved Columvi® (glofitamab-gxbm) for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) not otherwise specified or large B-cell lymphoma (LBCL) arising from follicular lymphoma, after two or more lines of systemic therapy. This indication is approved under accelerated approval based on response rate and durability of response in the Phase I/II NP30179 study. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Columvi will be available in the United States in the coming weeks. 

Patients with relapsed or refractory diffuse large B-cell lymphoma may experience rapid progression of their cancer ……………… press release continues

CLICK HERE to access the full press release

A boatload of biosimilars are now sitting in the approval circle since the first one was approved in early 2015.  Copies of just four market leaders, including Humira, Neulasta, Herceptin and Remicade, alone account for more than 20 approved biosim follow-ons.  And, the number of FDA biosims in the pipeline is expanding.

The article below details a recent report published by Cardinal Health that bears consideration saying, ”…. the resulting biosimilar versions are poised to change the biosimilar landscape as we know it.”  

The article below is timely as market resistance seems to be softening as payers begin to include more biosims on formulary.  Also, the number of biosims garnering interchangeability status (the holy grail) will allow for substitution where allowed…. four additional adalimumab biosimilar manufacturers are currently pursuing interchangeability.

The article also offers up some interesting prescribing trends within specialties….. with significant upticks in biosim confidence in Rheumatology, Gastroenterology, and Dermatology….. Oncology seems to be a holdout. On the payor front, formulary placement for the foreseeable future is likely to remain sticky due to pre-existing contracts between payers and manufacturers that have built in exclusivity / brand preference clauses that restrict biosims outright or force less preferred formulary placements.

Read on for more insights…..

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Will Humira Biosimilars Entry Cause a Wave or a Ripple?

By Gina Shaw, Pharmacy Practice News

With 2023 poised to be the year of the adalimumab (Humira, AbbVie) biosimilar wave, a new report focuses on the market dynamics that may affect how the new entrants are received by providers and other key stakeholders.

Although the report, issued by Cardinal Health, notes that adalimumab is “arguably the single largest pharmaceutical loss of exclusivity [LOE] event of all time,” and the resulting biosimilar versions are “poised to change the biosimilar landscape as we know it,” interviews with several experts suggest barriers remain. Whether it’s bundled contracts that give exclusivity to the innovator product or other exclusionary tactics, the experts noted that the true market outlook for adalimumab and other biosimilars remain a bit unclear.

One encouraging development, however, is the growing willingness of some providers to consider switching their patients to a biosimilar product, according to the Cardinal Health report. At least 75% of providers in all three relevant specialties surveyed—rheumatology, gastroenterology and dermatology—said they were somewhat comfortable with the prospect of prescribing a Humira biosimilar. Gastroenterologists appeared to be most comfortable, with 93% saying they were at least somewhat comfortable and 50% very comfortable; followed by rheumatologists (86% and 37%, respectively); and dermatologists (75% and 31%). Safety and efficacy as well as overall issues with switching were the most often cited concerns.

“Based on what we’ve observed in the trends within oncology, which had some of the first biosimilars, the differences in comfort level among these three specialties are probably just timeline related,” said Bruce Feinberg, MD, the vice president and chief medical officer of Cardinal Health. “The more exposure specialists have to these agents, [and] the more patients [are] under treatment, the more comfort they will have in prescribing them.”

Where We Are

The first adalimumab biosimilar, adalimumab-atto (Amjevita, Amgen), officially entered……

CLICK HERE to access the full article

Lots of angst between the FEDA and Pharma over drug pricing controls and price negotiations. 

There will be lobbying a’plenty by Pharma to mitigate what is already enacted and stem the tide of further price incursions.  The article below offers a scientifically based premise that there is a major flaw in the current pricing rules.

What is noteworthy is that the premise is based on the simple distinction of small vs. large molecule drugs. As noted, the recently enacted Inflation Reduction Act draws a distinction between large and small molecule medicines setting price control provisions to kick in  for small molecule medicines after 9 years (following FDA approval) vs. 13 years for large molecule biologics. 

Fowl most cruel crieth Pharma!

Why the angst? 

The developers say that they will lose the incentive to invest in post-approval R&D and patients will lose access to potential new treatments. It is here that the FEDS will start to push back saying that post approval R&D is not that costly….. especially if the original approval was for an orphan condition (that incidence is very high) which garnered valuable marketing benefits such as extended exclusivity. The FEDS will also likely push pack on the argument offered below that the developers will likely tend towards large molecule formulae because of the longer 13-year exemption from price controls.

The accountants can do the cost analyses but there is a good  likelihood that a small molecule candidate with a shorter price exemption period will generate more $$s because “patients prefer small molecule medicines that usually come in the form of pills over biologic injections or infusions by a wide margin.” And….. small molecule drugs “are generally easier to make at scale and nearly always less expensive for patients. Importantly, small molecule medicines are more easily genericized than biologics, leading to greater adoption and lower costs.”

So…. On which side of the argument do you lean?

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The IRA’s nonsensical distinction between small- and large-molecule drugs

Thanks to advances in science, notably the completion of the Human Genome Project 20 years ago, we are able to treat diseases more effectively than ever before. Researchers have more sophisticated tools not only to identify new drug targets, but also to make drugs to hit those targets. A wide variety of therapeutic agents help make these drugs, including chemically synthesized small molecules; larger molecules made of amino acids like peptides, proteins, and antibodies; and nucleic acids like RNA or DNA. Choosing among these approaches requires looking at the target attributes. Is it inside the cell or outside? Is the target an enzyme, a gene, or a lipid particle? Do we need to block the target or stimulate it? Science is — and should be — the guide to determine the optimal approach for patients.

That’s why, to researchers like me, a provision in the recently enacted Inflation Reduction Act is puzzling. For no clear reason, it draws a distinction between large and small molecule medicines. …………….. continues

CLICK HERE to read the full article

The FDA recently approved a new extended release oral-suspension therapy, Lumryz (sodium oxybate) from Avadel Pharmaceuticals, for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. Lumryz is taken once daily at bedtime.

Lumryz was approved with a black box warning for Central Nervous System Depression as well as Potential Abuse & Misuse. Lumryz is the sodium salt of gamma-hydroxybutyrate (GHB). Abuse or misuse of illicit GHB is associated with CNS adverse reactions, including seizure, respiratory depression, decreased consciousness, coma, and even death. A restrictive REMS has been imposed for the therapy.

CLICK HERE for prescribing information

Avadel confirmed that Lumryz will launch at $64.67 per gram.  Based on the highest daily dosing option (nine grams), the annual cost would top out at $212,440 per year.  This pricing is comparable to that of a competing product, Xywav, from Jazz Pharmaceuticals.

Given its cost and moderately high prevalence of ~140,000 in the USA, is is expected that Lymryz will be available only through specialty pharmacy limited distribution.

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Avadel Pharmaceuticals Announces U.S. Commercial Launch of LUMRYZ (sodium oxybate) for the Treatment of Cataplexy or Excessive Daytime Sleepiness in Adults Living with Narcolepsy

-LUMRYZ is the first and only once-at-bedtime oxybate for people living with narcolepsy

-Once-nightly dosing regimen of LUMRYZ has been found by FDA to provide a major contribution to patient care over all twice-nightly oxybates

DUBLIN, Ireland, June 05, 2023 (GLOBE NEWSWIRE) — Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, announced today that LUMRYZ is now commercially available. LUMRYZ is an extended-release formulation of sodium oxybate indicated to be taken once at bedtime for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy.

“We are proud to announce that LUMRYZ is commercially available through both our RYZUP™ patient support program and our specialty pharmacy network for patients living with narcolepsy who have been waiting for over two decades for a single dose treatment option that provides the opportunity for an uninterrupted night sleep,” said Greg Divis, Chief Executive Officer of Avadel. “LUMRYZ represents a new generation of oxybate treatment the FDA deemed clinically superior to all twice nightly oxybate treatments. The Avadel team is fully prepared to execute our commercial strategy and deliver LUMRYZ to the $3 billion plus once-at-bedtime oxybate market.”

LUMRYZ is the first and only U.S. Food & Drug Administration (FDA) approved once-at-bedtime oxybate for people living with narcolepsy. The commercial strategy for LUMRYZ includes an extensive patient support program, RYZUP, which is designed to provide support and education to patients who have been prescribed LUMRYZ and to assist them in gaining access to their medication. More information about the RYZUP patient support program can be accessed at ryzupsupport.com.

LUMRYZ was granted FDA approval based on positive results from the pivotal Phase 3 REST-ON clinical study completed in March 2020. In the REST-ON Phase 3 trial, once-at-bedtime LUMRYZ demonstrated highly statistically significant (p<0.001) and clinically meaningful improvement compared to placebo across all three co-primary endpoints (Maintenance of Wakefulness Test, Clinical Global Impression-Improvement and mean weekly cataplexy attacks) for all three doses evaluated, 6, 7.5 and 9 grams.

LUMRYZ was granted a seven year period of Orphan Drug Exclusivity as a result of the FDA finding LUMRYZ to be clinically superior to all first generation oxybate products. In particular, FDA found that LUMRYZ makes a major contribution to patient care over currently available, twice-nightly oxybate products by providing a once-nightly dosing regimen that avoids nocturnal arousal to take a second dose.

About Avadel Pharmaceuticals plc

Avadel Pharmaceuticals plc (Nasdaq: AVDL) is a biopharmaceutical company focused on transforming medicines to transform lives. Our approach includes applying innovative solutions to the development of medications that address the challenges patients face with current treatment options. Avadel’s commercial product, LUMRYZ, was approved by the U.S. Food & Drug Administration (FDA) as the first and only once-at-bedtime oxybate for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. For more information, please visit www.avadel.com.