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FDA Approves Oral Solution for Rare Liver Condition – Livmarli

…………catching up on FDA approvals

The FDA recently approved a new oral solution, Livmarli (maralixibat) from Mirum Pharmaceuticals, Inc, indicated for the treatment of cholestatic pruritus in patients 3 months of age and older with Alagille syndrome (ALGS) and also for the treatment of cholestatic pruritus in patients 5 years of age and older with progressive familial intrahepatic cholestasis (PFIC).  Livmarli is an ileal bile acid transporter (IBAT) inhibitor. It was granted Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC.

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in those affected. Signs and symptoms of PFIC typically include severe itching, jaundice, failure to thrive, and liver failure. The disease is estimated to affect fewer than 4,000 individuals in the US.

Published pricing for Livmarli oral solution (9.5 mg/mL) is about $56,240 for a supply of 30 milliliters.

Mirum did not announce plans for distribution. Given the small patient base, high cost and oral weight based dosing it is likely to be available through exclusive distribution.

CLICK HERE to access prescribing information


Mirum Pharmaceuticals’ Livmarli Receives FDA Approval for Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis

CLICK HERE to access the press release


CGT Driving New Programs at WAGS

Our last few Reports focused on approvals and developments in the Cell and Gene Therapy (CGT) categories. A recent press release from a major national pharmacy chain would seem to reinforce the notion that CGT is the newest best thing since sliced bread.

Walgreens is going through a major remake that the company says is reflective of where the market is and, more importantly, where it is going. They thought it was going towards walk-in medical care which is now in ‘rethink’ mode which is a Report for another day.   But the strategists at WAGS now see CGT as a better bet….. one that is more aligned with their legacy pharmacy model and where the rare/orphan/ultra-orphan market appears to be heating up fast….. and where you need to be a serious player to maintain its ‘creds’ in the marketplace.

According to the press release, “Effective August 1, 2024, AllianceRx Walgreens Pharmacy will become Walgreens Specialty Pharmacy.” While this is a clean-up on its fractionated branding, WAGS is also planning on investing in CGT in a tangible way by opening a “Gene and Cell Services Pharmacy and Innovation Center – a dedicated 18,000-square-foot center in Pittsburgh, PA, with services and capabilities for these emerging therapies, including innovative solutions for managing the complexity of the supply chain, logistics and financing as well as clinical and social needs management to ensure success for patients and partners.” 


Walgreens Launches Gene and Cell Services as Part of Newly Integrated Walgreens Specialty Pharmacy Business

Walgreens is expanding its specialty pharmacy services and investing in its capabilities as the company further grows its core pharmacy business to improve patient outcomes and provide greater value to payers and partners.

CLICK HERE to read the full press release


CGT…… What’s in Your Future?

Today we are pleased to offer a review of an article from which just about anyone working in the specialty pharmacy segment might learn something…. or even a lot.

The topic is cell and gene therapy (CGT)….. which is getting increasingly greater attention given recent approvals and a jaw dropping pipeline.

Can you define cell therapy vs. gene therapy? 

Even now, many would struggle to clearly differentiate between them.

Here’s what the article says……

  • Gene therapy is defined as the repair, replacement, or deactivation of dysfunctional genes to reestablish normal function. 
  • Cell therapy is defined as the transfer of autologous (from oneself) or allogeneic (donor) cellular material into a patient for medical purposes.
  • To devlop a CGT product a target gene or specific type of cell is identified to determine whether that gene or cell can be modulated directly within a patient’s own body (known as in vivo treatment), or if genetic or cellular material must be extracted from the body, modified, grown externally, and then transferred back into the patient (known as ex vivo treatment).
  • Over the past decade+ the FDA has approved more than 30 cell, cord blood, and gene therapy products (see list in the article).
  • Most investigational CGT products will continue to target oncologic conditions and rare diseases.
  • More than 2,000 CGT products globally are in various stages of clinical development. and…. as we have all surely noticed by now,
  • CGT therapies are among the most expensive medications in the world…….5 of the 10 most expensive drugs of 2023 were gene or cell therapies….. keeping health insurance companies awake at night worrying how to pay for them.

A reading of the full article will fill in much more detail of the current and, most importantly, give insight into the CGT ‘cures’ that one day are likely to reshape the legacy SP market. 


Cell and Gene Therapies: A Review of Current and Future Treatments

CLICK HERE to read the full article


FDA Approves Another Co$tly Gene Tx – Lenmeldy

The rate of FDA approvals of gene therapies is picking up….. at a pace not too dissimilar to the bevy of biosimilars we’ve witnessed in recent years. The newest gene therapy is Lenmeldy (atidarsagene autotemcel) from Orchard Therapeutics. Lenmeldy is the first FDA-approved gene therapy that is indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). 

MLD is caused by a mutation on the ARSA gene, resulting in a deficiency of the enzyme arylsulfatase A (ARSA). This deficiency causes sulfatides to build up in cells and damage the central and peripheral nervous systems. As a result, patients experience a loss of motor and cognitive function and early death. MLD, which has no cure, is estimated to affect one in 40,000 individuals in the United States.

Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own hematopoietic (blood) stem cells (HSCs), which have been genetically modified to include functional copies of the ARSA gene. The stem cells are collected from the patient and modified by adding a functional copy of the ARSA gene. The modified stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow.   The appropriate dose is calculated based on the child’s weight at time of Lenmeldy infusion using the instructions provided by the manufacturer. The processed dose is shipped to the treatment center in liquid nitrogen at less than -130°C (-202°F).

Lenmeldy now holds the austere position of being the most expensive therapy sold in the US. Orchard set the wholesale acquisition cost for Lenmeldy at $4.25 million.

CLICK HERE for prescribing information


Gene Therapy for MLD Gets FDA Approval

CLICK HERE to read the full article


FDA Approves Yet Another Humira Biosim – Simlandi

……. continuing to catch up on FDA approvals

The FDA recently approved a biosimilar, Simlandi (adalimumab-ryvk) from Alvotech and Teva Pharmaceuticals, as a biosimilar to Humira (adalimumab). Since there are already nine….. yep count ‘em….. other adalimumab biosims already approved, is there anything noteworthy to say about Simlandi…. YES!

The noteworthy thing about this approval is that Simlandi was approved with the coveted interchangeable designation. So, the ability for pharmacist substitution without physician approval in many states gives Simlandi a tangible marketing edge. However, the FDA had already approved Pfizer’s Abrilada (adalimumab-afzb) and Boehringer Ingelheim’s Cyltezo (adalimumab-adbm). The more the merrier?

But wait!…. Simlandi is high-concentration as well as citrate-free giving it a slight marketing edge over its interchangeable competitors….. as well as the rest of the pack.


FDA Approves Simlandi, Third Interchangeable Humira Biosimilar

CLICK HERE to read the full press release


Specialty Pharmacy Leader Board for 2023

There were no big surprises in the recently published list of the Top 15 specialty pharmacies nationally. That is if you only look at the ranking order and are not looking at revenues….. which continue to grow and grow generating about $242 billion in overall sales in 2023.

Here is the recently published list…….

Top 15 specialty pharmacies by 2023 revenue

Key takeaways—–

  • Two in five accredited specialty pharmacies are owned by hospitals and other healthcare providers
  • The nation’s three biggest pharmacy benefit managers accounted for 67% of specialty pharmacy revenue in 2023. 
  • Hospitals and health systems are the fastest-growing participants in this market, partly because of drugmakers’ restrictions regarding the 340B program, the report said. 
  • This year’s list included “Other retail, mail, long-term care and specialty pharmacies” as a group that  garnered over $37 billion of the total ~$242 billion or about 15%.

1. CVS Specialty (CVS Health) — $73.3 billion

2. Accredo/Freedom Fertility (Cigna/Evernorth/Express Scripts) — $59.5 billion

3. Other retail, mail, long-term care and specialty pharmacies — $37.4 billion

4. Optum Specialty Pharmacy (UnitedHealthGroup/OptumRx) — $32.3 billion

5. AllianceRx Walgreens Pharmacy/Walgreens stores — $8.4 billion

6. CenterWell Specialty Pharmacy (Humana) — $6.2 billion

7. Onco360 Pharmacy/CareMed Specialty Pharmacy (BrightSpring Health Services) — $4.6 billion

8. PANTHERx Specialty Pharmacy — $3.6 billion

9. Walmart Specialty Pharmacy/Walmart stores — $3.4 billion

10. Kroger Specialty Pharmacy/Kroger stores — $3.2 billion

11. Acaria Health (Centene) — $2.9 billion

12. Biologics/RxCrossroads (McKesson) — $2.2 billion

13. SenderraRx — $2 billion

14. AHF Pharmacy (AIDS Healthcare Foundation) — $1.9 billion

15. Lumicera Health Services (Navitus Health Solutions) — $1.5 billion

CLICK HERE to access the full article


FDA Approves 2 Biosimilars…in one — Jubbonti & Wyost

The FDA recently approved a ‘two-fer’…. yep, TWO identical biosimilars with the approval of Jubbonti (denosumab-bbdz) and Wyost (denosumab-bbdz) from Sandoz, Inc. They are the same formulations, and both received the coveted interchangeable designation. They are the first interchangeable biosimilars for RANKL inhibitors. Both therapies are available for subcutaneous administration. 

Jubbonti is indicated for the following (which are also currently approved for Prolia.)

  • postmenopausal women with osteoporosis at high risk for fracture;
  • increasing bone mass in men with osteoporosis at high risk for fracture;
  • glucocorticoid-induced osteoporosis in men and women at high risk for fracture;
  • increasing bone mass in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer; and
  • increasing bone mass in women at high risk for fracture receiving adjuvant aromatase inhibitor therapy for breast cancer.

Wyost is indicated for the following (which are also currently approved for Xgeva.)

  • prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors;
  • treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity; and
  • treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy.

CLICK HERE to access prescribing information for both therapies.

Sandoz has yet to confirm pricing. 

Similarly, distribution details for either/both therapies have not been disclosed.

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