JUNE 2023

THE VESSEL

BDA COMMUNITY EVENTS ~ Join the fun!

Did you miss our Pub Crawl? Our Night at the Joe? Or any of our educational dinners?

Don’t worry we have plenty of events in store! Plan to join us for our Annual Cornhole Tournament on August 19 at Lionheart on the Green, or our Annual Meeting on September 24 at Dave and Busters. And you have the whole summer to pull together your team for our UNITE Walk on October 14 at the Schodack Town Park. Get ready to dress down since this year our theme is Pajama Party!

And that’s just the beginning! We are also hosting a Women’s Retreat and an Insurance Education Day in November. Details of these events will be coming soom, so keep watching our website and Facebook page or follow us on Twitter and Instagram.

Our partners in the pharma industry continue to provide us with educational dinners as well. We hope to see you at one of these events soon.

The Board of Directors of Hemophilia Federation ofAmerica (HFA) unanimously selected Dan Kelsey as HFA’s new Chief Executive Officer (CEO) onApril 7, 2023. Dan will join the HFAteam on Wednesday,April 12 and attend their annual Symposium in Orlando, Florida.

Dan was first introduced to the bleeding disorders community early in his nonprofit career when he worked on database projects for Hemophilia of Indiana and the Indiana Hemophilia and Thrombosis Center while employed at the Indiana State MedicalAssociation in the areas of membership and payer advocacy. He also worked with the Indiana Medicaid program as a field representative and healthcare consultant in compliance, accounts receivable reviews, and various commercial payer issues. Dan has had vast experience working with statewide physician groups and associations over the past two decades.As previous Deputy CEO to a national physician specialty association and CEO of a national association focused on medical imaging and leadership, Dan brings a wealth of knowledge and experience to every facet of the organization.

“I am honored to be chosen as HFA’s next CEO. This team represents the values, collaboration and dedication required to strengthen the organization and lead by example as a patient advocacy group for rare diseases,” says Dan Kelsey. “I look forward to bringing a fresh perspective and new technologies to the work in order to deliver first-in-class outcomes for the bleeding disorders community.”

“Dan brings over 20 years of medical membership association, nonprofit and data systems experience to HFA. He has successfully led other organizations through key transitions and has inspired technological innovation,” says Luke Runion, Chair of HFA. “Dan currently serves as Chair of theAmerican Society ofAssociation Executives HealthCareAdvisory Subcommittee, which gives him unique insight into the issues impacting nonprofit organizations like HFA. The HFABoard of Directors is delighted to have Dan lead HFAand its staff in support of the bleeding disorders community.”

HFAis a community-based, patient-centered organization that advocates for the needs of families living with debilitating bleeding disorders. The primary goal of HFAis to advocate for safe and effective therapies, quality, affordable health coverage and better quality of life for all persons with bleeding disorders.

Here is a round-up of what’s happening in Washington~

Bilingual Resources

CMS published new resources (English and Spanish language versions) explaining consumer protections against unexpected out-of-network medical bills. The federal No Surprises Act protects people from unexpected bills for (a) out-of-network emergency room care, (b) non-emergency care received at an in-network facility, and (c) air ambulance services.

Medicaid Rights

The U.S. Supreme Court (Health and Hospital Corporation of Marion County, Indiana v. Talevski) ruled that individuals can sue under the federal civil rights statute to enforce their rights under the Medicaid program. Patient advocates including HFA and NHF had filed an amicus brief in the case and applauded the Court’s ruling.

Preventative Services

HFA and NHF joined with 14 patient groups in another amicus brief in Braidwood Management v. Becerra, litigation defending consumers’ rights to no-cost, evidence-based preventive services under the Affordable Care Act. The amicus brief demonstrates that preventive services save lives and are cost-effective, and outlines examples of the cost-free services that would be lost for many patients if the lower court’s adverse decision is upheld.

Insurance Utilization Report

KFF, the noted health policy research outfit, published an initial report on findings from a comprehensive study of consumer experiences with health insurance. KFF found, among other things, that: most insured adults give their own health insurance positive ratings (though people with more health needs gave lower ratings); despite those generally positive ratings, most insured adults report experiencing problems when they use their health insurance; affordability of premiums and out-of-pocket spending are a concern for about half of all privately insured consumers, with 4:10 adults skipping or delaying care due to cost; and insured adults overwhelmingly support policies to make insurance simpler to understand and use.

Prior Authorization Under Medicare Advantage

On June 21, 2023, 230 House members and 61 Senators wrote to the U.S. Centers for Medicare and Medicaid Services urging CMS to finalize rules (proposed last December) that would require Medicare Advantage plans to modernize and streamline the prior authorization process. The lawmakers called for: real-time prior authorization for routine care; a 24-hour deadline for “urgently needed care”; and more transparency around plans’ prior authorization metrics.

Consumer Protections for Employees of Small Businesses

The House Education and Workforce Committee advanced two partisan bills pitched as measures that would expand insurance options for small businesses. HFA and allied patient advocates voiced concerns that both bills would sidestep consumer protections and coverage requirements under the ACA.

HELP Co-Pays Act

This bill requires health insurance plans to apply certain payments made by or on behalf of a plan employee toward a plan’s cost-sharing requirements. Specifically, a plan must third-party payments, financial assistance, discounts, product vouchers, and other reductions in out-of-pocket expenses toward the requirements. The bill was introduced on February 6, 2023 and currently has 57 co-sponsors, including Yvette Clarke (NY9), Joseph Morelle (NY25), Daniel Goldman (NY10), and Jerrold Nadler (NY12). This bill remains our highest legislative priority.

Industry Update

BioMarin

On June 29, BioMarin announced that the United States Food and Drug Administration (FDA) approved ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. ROCTAVIAN was first approved by the European Medicines Agency in August 2022.

The FDA approval is based on data from the global Phase 3 GENEr8-1 study, the largest Phase 3 trial of any gene therapy in hemophilia. Of the 134 patients who received ROCTAVIAN in the study, 112 patients had baseline annualized bleeding rate (ABR) data prospectively collected during a period of at least six months on FVIII prophylaxis prior to receiving ROCTAVIAN. The remaining 22 patients had baseline ABR collected retrospectively. All patients were followed for at least 3 years. The majority of study participants continued to respond to treatment through year three and beyond, without supplemental use of regular prophylaxis.

BioMarin will continue to monitor the long-term effects of treatment with an extension study that will follow all clinical trial participants for up to 15 years, as well as postapproval studies to follow those dosed in a real-world setting for 15 years or more.

Pfizer

On June 27, Pfizer announced that the FDA has accepted its application for Biologics License Application for gene therapy (fidanacogene elaparvovec) for the treatment of adults with hemophilia B. In parallel, the European marketing authorization application (MAA) for fidanacogene elaparvovec has also been accepted and is under review by the European Medicines Agency (EMA). The FDA will be reviewing efficacy and safety data from the company’s Phase 3 BENEGENE-2 study and is expected to act by the second quarter of 2024.

An experimental Pfizer hemophilia drug, marstacimab, that takes a new approach to treating the blood clotting disorder has met the main goal of a pivotal study, setting the stage for regulatory submissions planned for later this year.

Marstacimab, is being evaluated in hemophilia A as well as the less common hemophilia B. Standard of care for both forms of hemophilia includes clotting factor treatments,

infusions of the clotting proteins that patients lack. But these therapies don’t last long in the body, so they must be administered every two-to-three days. Pfizer’s once-weekly injectable drug is a non-factor therapy.

Hemophilia A patients already have a non-factor drug option Hemlibra. That drug is an antibody designed to mimic the function of factor VIII. Its FDA approval covers the prevention of bleeding episodes. Marstacimab is also an antibody, but the Pfizer drug works in a different way. It’s designed to block tissue factor pathway inhibitor (TFPI), an anticoagulant protein in the body that inhibits the start of the blood clotting process. According to Pfizer, by targeting the Kunitz-2 domain of this protein, marstacimab may help reestablish the balance between bleeding and blood clot formation.

Novo Nordisk

Novo Nordisk is in the anti-TFPI chase with its antibody, concizumab, designed to treat both hemophilia A and B. Last month, the FDA rejected an application seeking approval, asking for more information about the monitoring and dosing of patients, the company disclosed in first quarter 2023 financial report. The FDA also asked for additional information about the drug’s manufacturing process. Concizumab is already approved in Canada for treating hemophilia B with inhibitors and is currently under regulatory review there for hemophilia A with inhibitors.

Hemab Therapeutics

Hemab Therapeutics, a clinical-stage biotechnology company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, announced results from preclinical research of HMB-001 to treat factor VII (FVII) deficiency. HMB-001 is bispecific antibody that binds and stabilizes existing factor VIIa (FVIIa) with one antibody “arm” and TLT-1 on activated platelets with the other “arm”. This allows for accumulation of FVIIa in the body, recruitment of FVIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage. HMB-001 was designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia (GT) with potential for other debilitating rare bleeding disorders, including factor VII deficiency.

CSL Behring

CSL Behring announced on June 20, that the first patient in

the United States has received HEMGENIX® (etranacogene dezaparvovec-drlb) for hemophilia B. HEMGENIX is the first and only gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes.

CSL Behring is providing ongoing training to centers who may administer HEMGENIX and encouraging long term data collection through the ATHN registry.

Vega Therapeutics

Vega Therapeutics, Inc., announced in May that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for VGA039 for the treatment of von Willebrand disease (VWD).

VGA039 is a first-in-class antibody therapy with a novel mechanism of action that modulates Protein S – a key cofactor involved in thrombin generation during both the initiation and propagation phases of coagulation. By promoting thrombin generation through targeting Protein S, VGA039 addresses a fundamental mechanism of clot formation in VWD and, as a subcutaneously selfadministered antibody therapy, has potential to transform VWD treatment.

DDAVP & Stimate Update

Nearly three years after Ferring Pharmaceuticals suspended production of nasal sprays that boost blood clotting in people with Type 1 von Willebrand disease and hemophilia A, it remains unclear if or when – the company will resume production. CSL Behring has declined to serve as their US distributor.

The production was suspended due to a packaging seal problem that led to evaporation and potentially dangerously high concentrations of the active ingredient, desmopressin.

Desmopressin is a synthetic form of vasopressin, a naturally occurring hormone with a wide range of effects that influence blood pressure, blood volume, urination and kidney function. Vasopressin can also increase the levels of von Willebrand factor and factor VIII. Desmopressin is the active ingredient in DDAVP nasal spray and Stimate. The two brand-names tend to get used interchangeably, although Stimate is actually a higher-concentration product. Patients who previously used DDAVP nasal spray or Stimate have been prescribed clotting factor instead, which is

more costly and requires intravenous infusion. In addition, clotting factor can lead to inhibitor development in patients with hemophilia A, adding to the difficulty and cost of care.

To bridge the gap, the Hemophilia Alliance, a trade association for many of the federally funded hemophilia treatment centers, turned to STAQ Pharma, a 503B outsourcing facility, which was able to put desmopressin into production in 2021 in the Stimate-level concentrations, after the FDA declared desmopressin a drug in short supply, a prerequisite for outsourcing approval.

STAQ Pharma is producing the drug for a wholesale cost of about $400 per vial, with roughly 6 actualizations, or uses, which is $80 less than the original Ferring cost, according to Joe Pugliese, president and CEO of the Hemophilia Alliance.

Still, it has been difficult to get payers to reimburse for this product, he said. “We've talked to the FDA several times about what we would have to do to get an abbreviated New Drug Application,” which would give the STAQ Pharma drug generic status and potentially improve the argument for payer coverage.

“Those conversations with the FDA have not been going quickly, which means that reimbursement and distribution are challenging at times. Support from pharmacy benefit managers has been mixed. Of course, hemophilia treatment centers and the smaller home care companies around the country have been purchasing the product and making it available to patients with good results,” Pugliese said. The uncertainty and interruption in supply has affected the size of the market for this drug despite its advantages over intravenous clotting factor.

“Some people moved to subcutaneous desmopressin acetate. Some people have moved to factor concentrates,” Pugliese said. However, about 2,000 vials of the STAQ Pharma nasal spray have been shipped since 2021.

“Getting the word out and then getting the patients connected with the product have been slow, but we continue to grow every month," Pugliese said.

Advocates for patients with hemophilia are also interested in STAQ Pharma producing supplies of another product for bleeding control called Amicar (aminocaproic acid), which Acorn Pharmaceuticals discontinued because of its Chapter 7 bankruptcy, announced in February 2023. There are other suppliers of this product, though, so the FDA may not want to add it to the drug shortage list.

How can you stay ahead of inflation? By getting a raise!

Could you use $1,000 to gain a skill or certification to help you get a job? HFA is offering adults, ages 18-64 the opportunity to apply for a $1,000 training grant to cover costs associated with acquiring technical skills or certification for career advancement. Examples include: project management, dog grooming, customer service, medical billing, web design, phlebotomy, real estate sales, SCRUM methodology, and more.

The applicant must be a person with a bleeding disorder between the ages of 18 to 64. Carriers are eligible. Past recipients of a Job Readiness grant cannot apply. Applicants cannot be HFA boardmembers, HFA staff, or immediate family members of the board or staff. Funds must be used for technical skills courses or certifications.

Applications must include: a brief statement of need; proof of a bleeding disorder; resume; and documentation of course cost. A letter of recommendation and completion of HFA’s Learning Central’s Career Planning course are both optional, but recommended.

Applications are processed as soon as they are receivedand will be available until all granys have been distributed, so be sure to apply today! Access the application and further information here.

We are now accepting applications to compete! You must be between 7 to 11 years of age and indicate which of the following sports you would like to compete: baseball, basketball, golf or swimming..Please submit a short essay on why you would like to compete and send to: beth.m@bdaneny.org

The New York state Legislature ended its 2023 session earlier this month. Here is a recap of the issues we have been following, provided to us by the New York State Bleeding Disorders coalition

The Coalition for Hemophilia B is now accepting applications for their Men’s Fall Retreat. Learn more about the program and apply to join here.

Contact Us

BDANENY Office

333 Broadway Suite 210 Troy, NY 12180 (518) 729-3577

BDANENY.org

memberservices@bdaneny.org

BDANENY Board of Directors and Staff President…………………….…………..…Bruce Weiner

Vice President…………………….………..Chris Huskie

Treasurer………………………………….....Cindi Keeler

Secretary……..…….………….…..Christina Mastrianni

Board Member…………………..Juliemar Abreu Boria

Board Member………..……...…....…Matthew Delaney

Board Member………………………..….Linda Peacock

Board Member……………..……………..Jessica Smith

Executive Director……………..…….….…..Beth Mahar

Operations Manager………………....Patricia Bengraff

Special Events & Social Media…..…......Melissa Deeb