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FDA Approves New Tx for Rare CDD – Ztalmy

The FDA recently approved a new oral suspension therapy, Ztalmy (ganaxolone) to treat seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. This is the first treatment for seizures associated with CDD and the first treatment specifically for CDD. Ztalmy was developed by Marinus Pharmaceuticals and is supplied as an oral suspension.

Patients with CDKL5 deficiency disorder (CDD) typically have infantile-onset epilepsy that responds poorly to previously available treatments. Symptoms include hypotonia (poor muscle tone), severe developmental and cognitive delays with little or no speech production, fine and gross motor impairment (including inability to walk for most patients), cortical visual impairment, behavioral abnormalities, and sleep and digestive difficulties. 

CDD is a rare disease with an incidence of between 1 in 40,000-60,000 live births, making it one of the most common genetic forms of epilepsy. This translates into ~6,500 lives in the US.
Marinus announced that Ztalmy will launch in Q3 pending DEA controlled substance approval. It will have a WAC of $133,000 per patient per year and will average $105,000 per patient per year after discounts.

Given the small patient opportunity and clinical complexity of the therapy it is very likely that access to Ztalmy will be limited to a specialty pharmacy.

CLICK HERE to access prescribing information


FDA Approves Another CAR-T Tx for Multiple Myeloma – Carvykti

The FDA recently approved a new infused therapy, Carvykti (iltacabtagene autoleucel), from Janssen Biotech, for the treatment of adult patients with relapsed or refractory multiple myeloma.   The therapy was developed in partnership with Legend Biotech. The partners will share marketing in the US.

Carvykti is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. Each dose is customized using a patient’s own T-cells, which are collected and genetically modified, and infused back into the patient.

Carvykti will compete in the market with another BCMA-targeted CAR-T drug approved a year ago, Abecma from 2seventy bio. Janssen announced that Carvykti will launch at a wholesale acquisition cost of $465,000 vs. Abecma’s $419,500 list price.

Carvykti was approved with an extensive BOXED WARNING as well as a restrictive access REMS program.

Janssen did not announce details on distribution or whether access would be through a specialty pharmacy distribution program.

CLICK HERE to read the full FDA press release


Limited Distribution Deals Confirmed

Announcements for newly approved specialty drugs often state that the product will be available through specialty pharmacy in limited distribution. However, the press releases rarely specify the specialty pharmacy(ies) selected as the designated partner(s).

Here are two LD deals that have been recently publicly confirmed subsequent to the approvals.

Soleo Health Selected as Limited Distribution Partner for VYVGART
January 24, 2022 — FRISCO, TX– Soleo Healthannounced today it was selected as a limited distribution partner for VYVGART™ (efgartigimod alfa-fcab) by argenx SE. VYVGART was approved by the U.S. Food & Drug Administration (FDA) on December 17, 2021 for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.

Soleo Health will manage and administer VYVGART intravenously in patients’ homes or its ambulatory infusion centers nationwide. Patients are supported through Soleo Health’s Myasthenia Gravis Therapeutic Care Management Center (TCMC). The TCMC is staffed with specially trained interdisciplinary teams, comprising experienced therapy care pharmacists, registered nurses, reimbursement specialists and patient care ambassadors dedicated to supporting patients living with myasthenia gravis.

PANTHERx Rare Selected as Exclusive Distribution Partner for FYARRO
Feb. 24, 2022 – PITTSBURGH, PA– PANTHERx Rare announces that it has been selected by Aadi Bioscience as the exclusive U.S. pharmacy distribution partner for new drug FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound). FYARRO, an albumin-bound mTOR inhibitor administered as an injectable suspension, is the first FDA-approved therapy for the treatment of advanced malignant perivascular epithelioid cell neoplasm (PEComa), an ultra-rare sarcoma.

Advanced PEComa is a rare, aggressive subset of soft tissue sarcomas for which there are no other approved therapeutic options. PEComas are composed of histologically and immunohistochemically distinctive epithelioid cells and most commonly arise from visceral, retroperitoneal, and abdominopelvic sites.


Will New FDA Rules Be Problematic for Specialty Pharmacy WDD and 3PL Business?

Over the past decade more and more specialty pharmacies obtained wholesaler/distributor (WDD / 3pl) licenses in addition to the traditional pharmacy license. Most of the therapies sold under those licenses are direct-to-provider for in office/clinic administration and commonly buy-and-bill. As you probably know, a majority of these meds are very high cost and are related to a limited / exclusive distribution program with the manufacturer.

The FDA is now moving to impose new rules that will supersede state regulations for these transactions, such as requiring national licensing, and ramped up operational standards and more. Legal experts on this matter believe that the proposed rule could have a significant impact on WDD and 3PL business operations. 

This is the time that SPs distributing meds under a state wholesale/distributor licenses to immediately assess all impacts to their business model and submit comments to FDA on the proposed rule changes by June 6, 2022 (Docket No. FDA-2020-N-1663).  You snooze you lose!

The FDA intends to conduct a webinar to provide an overview of the proposed rule to help stakeholders understand the proposed rules and prepare comments. 

The key provisions of the proposed rule include the following:

  • Definitions of additional terms to help clarify the requirements of the proposed rule and align with current industry practice and existing law, including “3PL activities”, “key personnel”, “minimal quantities”, “other logistics services”, “other than a consumer or patient”, “product”, and “wholesale distribution”;
  • National licensure and application requirements for WDDs and 3PLs, as well as the federal licensure review process;
  • Standards and procedures that set forth licensure denial, suspension, reinstatement, revocation, and voluntary termination, including notice and the opportunity to request a formal evidentiary public hearing under existing FDA hearing procedures;
  • Requirements for pre-license and post-license inspections;
  • Personnel requirements and qualifications necessary for licensure;
  • Surety bond requirements for WDDs;
  • Requirements to establish good storage practices, including personnel requirements and qualifications necessary for good storage practices compliance;
  • Requirements and standards for written policies and procedures to address a product’s sale, purchase, receipt, examination, security, storage, handling, inventory, shipment, transportation, disposition, and distribution;
  • Requirements, standards, and procedures for record keeping, document maintenance, and initial and annual reporting; and
  • Requirements, standards, and procedures for the FDA’s approval of third-party accreditation or inspection services or programs (“approved organizations” or “AOs”) to review the qualifications of 3PLs for licensure and conduct inspections of 3PL and WDD facilities, including the standards and procedures for organizations whose requests for approval are denied and for AOs whose approvals are suspended or revoked.

CLICK HERE to access the full Proposed FDA Rule

CLICK HERE to read the FDA Press Release including links to related documents 

FDA Publishes Proposed Rule on National Standards for The Licensure of Wholesale Drug Distributors and Third-Party Logistics Providers as Required by The Drug Supply Chain Security Act

March 11, 2022 — The US Food and Drug Administration (FDA) published its proposed rule National Standards for the licensure of Wholesale Drug Distributors and Third-Party Logistics Providers on February 4, 2022. The proposed rule sets national standards for the licensing of prescription drug wholesale distributors (WDDs) and third-party logistics providers (3PLs) with the goal of strengthening the security of the US drug supply chain. When finalized and effective, these licensing regulations will preempt state and local licensing standards, requirements, and regulations that are different from these federal requirements.
CLICK HERE to read the full article from the National Law Review


CPhT Launches Suite of Certifications for Rx Techs

Specialty pharmacies have seen margins shrink in spite of increasing volume. Many SPs turned to automation and clinically intuitive customer service software enabled SPs to improve speed to therapy and maintain quality of care standards. But overhead continues to be very people heavy. In short, bodies in the pharmacy and on the phone with customers are a big $$$ element of the delivery model.

It is not surprising, therefore, that SPs would look to their infantry in the pharmacy, pharmacy technicians, to shoulder a larger role in the delivery model. With tight clinical pharmacist availability and salaries rising, a certified pharmacy tech could be counted on to fill the gap. So, it should be no surprise that SPs interested in promoting their clinical quality would also want their techs to polish their clinical and operational expertise.

As noted in the article below, the CPhT has announced new course ware to enable pharmacy techs to obtain certifications in a variety of professional areas.

The certifications include the following:
– Advanced Certified Pharmacy Technician (CPhT-Adv),
– Board Certified Sterile Compounding Pharmacy Technician (BCSCPT),
– Board Certified Nonsterile Compounding Pharmacy Technician (BCNSPT).
Specialty certificates include:
– Technician Product Verifications (TPV)
– Medication History
– Controlled Substance Diversion Prevention
– Hazardous Drug Management
– Billing & Reimbursement
– Immunization Administration
– Point-of-Care Testing,
– Medication Therapy Management (MTM)
– Regulatory Compliance
– Supply Chain Management

Board of Pharmacy Technician Specialties Launches with Three Certifications and 10 Certificates to Advance Technicians’ Careers

New NPTA initiative offering three advanced certifications, including CPhT-Adv, and ten certificates providing increased accessibility and affordability to advance the careers of pharmacy technicians and enhance patient care and pharmacy practice

HOUSTON, Jan. 18, 2022 /PRNewswire/ — The Board of Pharmacy Technicians Specialties (BPTS), an independent initiative of the National Pharmacy Technician Association (NPTA), today launched three new advanced certifications for pharmacy technicians, including the CPhT-Adv, and ten specialty certificates. These new offerings will increase accessibility to credentials that will validate the skills and knowledge for pathways to advance the careers of pharmacy technicians, enhance patient care, and strengthen the workforce for pharmacies.

NPTA has long been the industry leader in advanced training programs and specialty certifications for pharmacy technicians, such as their sterile compounding program where NPTA has trained and certified more than 13,000 individuals. BPTS’s new certifications and certificates expand on NPTA’s mission to support, educate, and advocate for pharmacy technicians.

Exams will be offered at just $49 and be open to individuals certified through either NHA or PTCB to ensure accessibility to career-advancing credentials to all certified pharmacy technicians. The exam fee will include one additional retake at no charge, providing significant value compared to industry alternatives. BPTS has also set eligibility requirements to be more closely aligned with those expected of board-certified pharmacists, where individuals are encouraged but not required to take specific training programs, so long as they have adequate work experience.

“The responsibilities placed on pharmacy technicians to respond to increased patient needs are greater than ever,” said Mike Johnston, CEO of the National Pharmacy Technician Association. “With the launch of BPTS and these new offerings, all certified pharmacy technicians will now have the ability to demonstrate their advanced skills and competencies, earn advanced credentials, and ultimately gain the recognition, respect and compensation they deserve.

Registration for the certifications and certificates is available now, and the first exams will be offered in mid-February. Pharmacy technicians may register today at


FDA Approves 34th Biosimilar

Lo and behold…… the FDA approved yet another biosimilar to Filgrastim.
This follow-on product joins two previously approved biosimilars to Neupogen and was christened Releuko (filgrastim-ayow) from Amneal Pharmaceuticals and Kashiv Biosciences. It is the first biosimilar for Amneal, which is in development of two additional biosimilars expected later this year.

Filgrastim-ayow is indicated for febrile neutropenia in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs, for expedited neutrophil recovery, related clinical sequelae and severe neutropenia.

However, Releuko comes with warnings and precautions such as fatal splenic rupture, acute respiratory distress syndrome, allergic reactions including anaphylaxis, myelodysplastic syndrome, thrombocytopenia, and serious reactions in patients with Sickle cell.

Releuko enters a competitive segment win the market against brand Neupogen and biosims Zarxio (approved 03/2015) and Nivestym (07/2018)and. Releuko pricing was not available at time of the announcement. By way of reference, the approved filgrastim biosimilars, both of which are available commercially, are priced at only ~15% discount to the brand.

Kashiv, Amneal Receive First FDA Approval for Their Filgrastim Biosimilar

March 3, 2022 — Releuko, a filgrastim biosimilar developed by Kashiv Biosciences and Amneal Pharmaceuticals, has received FDA approval, the first of 2022.

The FDA has approved Releuko (filgrastim-ayow), a filgrastim biosimilar referencing Neupogen developed by Amneal Pharmaceuticals and Kashiv Biosciences. The biosimilar marks the third filgrastim biosimilar to be approved for the US market.

The product is also the first biosimilar to receive FDA approval for the Kashiv and Amneal teams and represents the first FDA biosimilar approval of 2022. The companies said that they expect to launch the product during the third quarter of 2022.

“The US approval of our first biosimilar is a very significant milestone for Amneal. Biosimilars represent the next wave of providing access to affordable medicines in the United States. We are building a global biosimilars business by leveraging partner assets to start and then leveraging our own key capabilities over time. Our goal is to become a meaningful long-term player in biosimilars,” said Chirag and Chintu Patel, co–chief executive officers of Amneal, in a statement.

The biosimilar will be used in patients with cancer undergoing chemotherapy who are at risk for developing febrile neutropenia, a common complication related to chemotherapy where the level of neutrophils in a patients’ blood stream are too low.

“It is a proud moment for the Kashiv team and our partners at Amneal to have our first biosimilar, Releuko, approved by the US FDA. Kashiv is one of a few domestic companies to manufacture and launch a biosimilar in the United States. Kashiv aims to continue bringing high-quality biosimilars to the global markets over the coming years. I would like to extend a humble ‘thank you’ to our highly talented team, without whom this would not have been possible,” said Chandramauli Rawal, MBBS, chief operating officer for Kashiv, in a statement.

The companies are also working together to develop 2 other oncology biosimilars: a pegfilgrastim biosimilar referencing Neulasta and a bevacizumab biosimilar referencing Avastin. They said they hope to launch them in 2022 and that both are under review by the FDA.

Releuko was approved for administration for intravenous and subcutaneous use, either as single-dose vials or prefilled syringes, both of which come in a 300-mcg/mL dose and 480-mcg/1.6 mL dose.

Under the agreement between Kashiv and Amneal, Kashiv will be handling the US launch and the manufacturing. In January 2021, Amneal and Kashiv announced an agreement under which Amneal would acquire a 98% interest in Kashiv’s specialty pharmaceuticals division. Kashiv Biosciences is headquartered in Piscataway, New Jersey, but has facilities in Chicago, Illinois. Amneal is based in Bridgewater, New Jersey.

Kashiv Biosciences was originally established in 2006 as Therapeutic Proteins, according to founder and consultant for the company, Sarfaraz K. Niazi, PhD, a member of The Center for Biosimilars® Advisory Board. The company’s name was changed in 2014 to Adello Biologics and then to Kashiv Biosciences.

Skylar Jeremias, Center for BIosimilars


FTC Disappoints Most Specialty Pharmacies

Only the second and third tier specialty pharmacies are crying in their beers last week. It comes on the heels of the FTC Commission’s deadlocked vote to study PBM practices that have had hugely negative financial impacts on those pharmacies. The deadlock effectively stalls any action on the issue. By contrast, the nation’s largest specialty pharmacies, mostly owned and operated by PBMs, sat silent….. to no one’s surprise.

Much has been written about the unfairness of the practice of assessing DIR fees….. so we won’t recap the nitty-gritty details. Curiously, the same big box SPs have been able to exempt themselves from paying these same fees. While relief from paying the DIR fees would be a boon to the SPs now paying fees, the issue is simply too mysterious for the general public to comprehend and ferment activism. In short, the PBM lobby was successful in burying the issue…. at least for the moment.

We expect the fight to go on.
It is lucky we don’t have a pandemic and an invasion to distract attention from this issue…. oh wait!

FTC Votes Against Investigating PBMs’ Effects on Specialty Pharmacies

02/22/2022 — On Feb. 17, The Federal Trade Commission deadlocked on a vote to study pharmacy benefit managers (PBMs) and how their practices affect independent and specialty practices, effectively killing a probe that pharmacy and patient advocates have said is long overdue.

The 2-2 vote came after nearly two hours of testimony from numerous independent pharmacy representatives, who said PBM tactics—such as low reimbursement rates and affiliations with large retailers—have pushed many small practices out of business. No one testified on behalf of PBMs.

“After hearing hours of testimony by community pharmacists and patients, all of whom painted the same shocking picture about PBM abuse, and not a single witness there to defend the PBM industry, it is inexplicable that two members of the commission could vote against the study,” the National Community Pharmacists Association said in a statement. “We’re grateful to Chairwoman [Lina] Khan for bringing this issue to a vote. We will demand she do so again at the soonest possible opportunity. However, this is extremely disappointing.”

Pharmacy and patient advocacy groups have long called for the federal government to investigate PBMs, saying they have contributed to prescription price increases and fewer marketplace options.

For example, because PBMs set formularies, patient options for medications are limited, pharmacists say. They have also criticized spread pricing, and the growing trend among PBMs to move claims processing from the medication side to the pharmacy side, requiring more white and brown bagging.

In a blog post last week, JC Scott, the president and CEO of the Pharmaceutical Care Management Association, which represents U.S. PBMs, said PBMs “are widely recognized for negotiating directly with pharmaceutical companies to lower drug costs.”

“Through expertise, data, and technology, PBMs pave the way for a seamless pharmacy experience helping patients to get and stay on their medications and through the process, lead healthier lives,” he said.

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