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FDA Approves New Tx for gMG – Zilbrysq

…..catching up on FDA Approvals

The FDA has approved a new subcutaneous therapy, Zilbrysq (zilucoplan) from UCB, for the treatment of adults with generalized myasthenia gravis.  Zilbrysq is the first once-daily subcutaneous, targeted C5 complement inhibitor for gMG. It is the only once-daily gMG-target therapy for self-administration.

gMG is a rare autoimmune disease with a global prevalence of 100–350 cases per every 1 million people. Symptoms of the disease include severe muscular weakness, drooping eyelids, difficulty with swallowing, chewing and talking, as well as life-threatening weakness of the respiratory muscles.

Zilbrysq was approved with a Black Box Warning for serious meningococcal infections.

CLICK HERE to access prescribing information 

UCB has not yet confirmed pricing for Zilbrysq.

Given that Zilbrysq is a self-administered, sub-q therapy with a Black Box Warning, it is expected that it will be channeled through specialty pharmacy limited distribution.

CLICK HERE to access the full UCB press release


Start Tracking Outcomes in Specialty Pharmacy Now

Specialty pharmacies produce a lot of reporting…… but, the scope of this reporting may be missing the mark in the evolving competitive marketplace. The real challenge in this new environment will be all about outcomes. The challenge will be not in analyzing and reporting outcomes but, rather, what interventions were used to improve outcomes. Therein lies the rub.

Two articles were recently published that drilled down into the deeper layer of outcomes reporting by forcing health care providers to demonstrate how they are proactively changing outcomes through targeted interventions. Note that we say ’health care providers’, not just the pharmacy. The way to proactively impact outcomes will require participation by multidisciplined, non-siloed teams. Hospital systems are using this mantra to promote their differentiated specialty pharmacy model. 

One article lays out a roadmap to consider to navigate these new waters starting with a three-step process

  • Environmental scan (developing a list of all potential measures, include quality measures / guidelines for each disease state).
  • Expert engagement (rely on subject matter experts to identify the most important measures.)
  • Implementation (include front line and informatics expert members in a dedicated team.)

Develop a strategy to produce better outcomes documentation include;

  • Determine the value story
  • Identify the outcomes that demonstrate value (not just numbers). 
  • Define outcome goals
  • Create targeted data elements to document outcomes
  • Collect the data to report meaningful outcomes
  • Report outcomes to key stakeholders 

The kind of outcomes reporting suggested will be hard…… no shortcuts here.

However, it may be the key differentiator that the market will start to demand.


CLICK each title to access the full article

Start Tracking Outcomes in Specialty Pharmacy Now

Using Data-Driven Monitoring Tools Boosts SP Outcomes


FDA Approves Two Gene Therapies for Sickle Cell on Same Day

Another milestone has been passed in the advancement of specialty pharmacy therapies. The FDA has approved not one, but two, gene therapies for a major disease, Sickle Cell Anemia. Most noteworthy is that one of the approvals offers real proof of concept for CRISPR gene-editing technology.

FIRST….. The FDA has approved a unique therapy, Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and CRISPR Therapeutics, indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). Casgevy is an autologous genome edited hematopoietic stem cell-based gene therapy. 

Sickle cell affects an estimated 100,000 people in the United States. The disease can impact any race, however, the black community is the most affected with 90+% of all diagnosed cases. In sickle cell disease, the body makes flawed, sickle-shaped hemoglobin, impairing the ability of red blood cells to properly carry oxygen to the body’s tissues.

The process for Casgevy therapy requires significant preparation. Stem cells harvested from the patient’s bone marrow are sent to manufacturing facilities where they are edited using CRISPR/Cas9 technology. Once the cells are incubated, they are infused back into the patient during a month-long hospital stay.

Second….. The FDA also approved another therapy, Lyfgenia (lovotibeglogene autotemcel) from bluebird bio. This gene therapy is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene. Lyfgenia is also an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events.

The key difference is that Casgevy introduces a manufacturer, gene-edited therapy vs. Lyfgenia which is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene that enables production of adult hemoglobin that does not form into the crescent shape associated with the disease.

Refer to Prescribing Information for each therapy for more detail on these complex processes especially around dosage and administration.

CLICK HERE to access Casgevy label 

CLICK HERE to access Lyfgenia label 

Neither company disclosed launch pricing at time of approval.

Neither company disclosed distribution plans at this time.

CLICK HERE to read the full press release


Limited Distribution Updates

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 five LD deals that have been publicly confirmed subsequent to their approvals.

Orsini Specialty Pharmacy to Distribute Adzynma 

Orsini Specialty Pharmacy has been selected by Takeda Pharmaceuticals as the exclusive specialty pharmacy partner for ADZYNMA (ADAMTS13, recombinant-krhn) for prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP). ADZYNMA is the first and only FDA-approved recombinant ADAMTS13 (rADAMTS13) enzyme replacement therapy for people 

Onco360 to Distribute Fruzaqla

Onco360 has been selected as a pharmacy partner by Takeda for Fruzaqla (fruquintinib an oral targeted therapy for certain adults with metastatic colorectal cancer (mCRC).  Fruzaqla is the first and only selective inhibitor of all three VEGF receptor kinases approved in the U.S. for previously treated mCRC regardless of biomarker status. Fruzaqla is a selective oral inhibitor of VEGFR-1, VEGFR-2, and VEGFR-3, which play a pivotal role in blocking tumor angiogenesis.

Biologics by McKesson to Distribute Truqap

Biologics by McKesson announced that it has been selected by AstraZeneca as a specialty pharmacy provider for Truqap (capivasertib), an orally administered drug, in combination with fulvestrant, for the treatment of adult patients with hormone receptor-positive, human epidermal growth factor receptor 2-negative locally advanced or metastatic breast cancer with one or more biomarker alterations (PIK3CA, AKT1, or PTEN).

Walgreens’ (WBA) AllianceRx Pharmacy to  Distribute XDEMVY

Walgreens Boots Alliance’s WBA wholly-owned subsidiary — AllianceRx Walgreens Pharmacy — will distribute Tarsus Pharmaceuticals’  XDEMVY (lotilaner ophthalmic solution) 0.25%. AllianceRx Walgreens is one of the four specialty pharmacies selected to distribute the FDA-approved treatment for Demodex blepharitis (DB), a highly prevalent eyelid disease.

Biologics by McKesson to Distribute Ogsievo

Biologics by McKesson has been selected by SpringWorks Therapeutics as a limited distribution specialty pharmacy for Ogsiveo (nirogacestat). Ogsiveo is a gamma-secretase inhibitor indicated for adult patients with progressing desmoid tumors who require systemic treatment.


FDA Approves Oral Tx for UC – Velsipity

…………..catching up on recent FDA approvals

The FDA recently approved a new ORAL therapy, Velsipity (etrasimod) from Pfizer, for adults with moderately to severely active ulcerative colitis (UC).  Velsipity is a once-daily, oral, sphingosine 1-phosphate (S1P) receptor modulator that selectively binds with S1P receptor subtypes 1, 4, and 5.

UC is a chronic, debilitating condition affecting about 1.25 million people in the United States. Symptoms often include chronic diarrhea with blood and mucus, abdominal pain, and urgency.  The therapy provides patients the opportunity to achieve steroid-free remission.

Velsipity has launched with a wholesale acquisition cost is $6,164 for 30-day supply or ~$75,000 annually. Pfizer did not disclose distribution details although, given its cost, it is highly likely to be dispensed only through specialty pharmacy limited distribution.

CLICK HERE to read prescribing information


U.S. FDA Approves Pfizer’s Velsipity for Adults with Moderately to Severely Active Ulcerative Colitis

CLICK HERE to read the full press release


CarelonRx Goes Digital

There has been scant news about any big moves by large national specialty pharmacies this year. Today, however, we read about a move by CarelonRx, the specialty pharmacy operated by Elevance Health.

Many of you may be scratching your heads trying to figure out the story behind these entities. We are happy to provide some relief.

In late 2022 Elevance Health acquired long-running BioPlus Specialty Pharmacy as it sought to expand its specialty pharmacy services. (BioPlus was one of the few mature SPs that had not been acquired and taken out of the independent SP column.) More significantly, Elevance is the insurer formerly known as Anthem Health….BioPlus was then integrated into Elevance’s – then branded – PBM, IngenioRx. 

We continue to ask….. Why are insurers keen on integrating SPs into their portfolios?

In a word, money….. and leverage (guess that’s two words). A well-run SP with a solid revenue stream is very profitable. Elevance appears poised to supercharge its market presence both ‘digitally’ and geographically which enables greater steerage of plan members into their specialty pharmacy and supercharge profit opportunity.  And…… PBMs love leverage over manufacturers. Being able to put large specialty purchases on the table goes a long way in manufacturer negotiations.


Elevance Health’s CarelonRx rolling out new digital pharmacy

Dec 5, 2023

The CarelonRx Pharmacy launch is one of several steps that the company is taking to improve the experience for members.

Elevance Health’s pharmacy benefit manager is launching a new digital pharmacy that aims to make it easier for members to track their prescriptions.

CarelonRx Pharmacy will launch on Jan. 1, and members will be able to connect with pharmacists via text, chat or phone around the clock. The platform will also allow them to check the price of medications for comparison and track their prescriptions throughout the order process, similar to how a customer may follow a pizza order.

……………………………….article continues

CLICK HERE to access the full article


FDA Approves New Combo Tx for Pompe – Pombiliti

………..catching up on recent FDA approvals

The FDA recently approved a new infused therapy, Pombiliti (cipaglucosidase alfa-atga) from Amicus Therapeutics, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy. 

Pombiliti is approved only in combination with Opfolda (miglustat), an oral drug that is to be administered approximately 1 hour before the infusion of Pombiliti. 

Pombiliti is a recombinant human GAA enzyme (rhGAA). Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood. Pombiliti / Opfolda will be distributed in tandem. 

About 5,000 to 10,000 people have been diagnosed globally with Pompe. The majority of patients on current standard of care decline after about two years. The previous standard of care has been Sanofi’s Lumizyme. 

The combo therapy launched at an annual price of $650,000 for a patient weighing about 70 kg.

Pombiliti will be distributed by Orsini Specialty Pharmacy according to a recent press release.

Pombiliti was approved with a black box warning.

CLICK HERE to access prescribing information

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