Denise Myshko is your editorial contact for the Formulary Watch. She joined Formulary Watch as editor is June 2021 from Oncology Live. Prior to that, she was managing editor of PharmaVOICE, a trade publication in the pharmaceutical industry. Email: dmyshko@mjhlifesciences.com

 Cimerli (ranibizumab-eqrn) as a biosimilar product interchangeable with Genentech’s Lucentis (ranibizumab). Coherus BioSciences plans to launch the ophthalmologic therapy in early October 2022.

Cimerli will be available in both 0.3 mg and 0.5 mg dosages and will be interchangeable for all of Lucentis’s indications, including neovascular age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, diabetic retinopathy, and myopic choroidal neovascularization.

“Retinal disease is a significant public health issue with certain conditions leading to vision loss or impairment. As a practitioner committed to the safety and well-being of patients, having an approved biosimilar product that is interchangeable with Lucentis — with a similar safety and efficacy profile — is great news for patients,” Peter K. Kaiser, M.D., professor of Ophthalmology at the Cole Eye Institute/Cleveland Clinic, said in a press release.

Cimerli belongs to the anti-VEGF therapy class of biologics. 

The interchangeable approval was based on the COLUMBUS-AMD study to confirm equivalent safety and efficacy to Lucentis. 

 this was a head-to-head study where Cimerli met its primary endpoint of change from baseline in best corrected visual acuity (BCVA) at week eight as compared with reference ranibizumab. Secondary endpoints included change from baseline in BCVA at 48 weeks, change from baseline in FCB retinal thickness at 48 weeks, safety and immunogenicity.

The overall safety and immunogenicity profile was comparable with Lucentis. Based on the totality of evidence, Cimerli demonstrates that clinical outcomes are expected to be the same for any given patient across all indications.

The first biosimilar with interchangeability status compared with its reference product is entitled to one-year of exclusivity of the interchangeability designation.

Coherus didn’t provide a price for Cimerli, but another Lucentis biosimilar, Byooviz, has a list price of $1,130 per single use vial, which is 40% lower than the current list price of Lucentis. Byooviz, developed by Biogen and Samsung Bioepis, launched in June 2022 but it doesn’t have an interchangeable approval. The FDA approved this biosimilar in September 2021 for the treatment of wet macular degeneration, macular edema following retinal vein occlusion, and myopic choroidal neovascularization.

Related: Biogen/Samsung Bioepis Launch the Biosimilar Byooviz

Articles

Coherus’ Cimerli has been approved to be interchangeable for all five indications, including age-related macular degeneration and diabetic retinopathy. It will be available in early October 2022.

FDA Approves First Interchangeable Biosimilar to Lucentis

Higher out-of-pocket costs contributed to lower patient adherence to oral cancer medications, according to a retrospective 

Journal of Managed Care + Specialty Pharmacy

. Investigators found that adherence was less than optimal and was worse for patients with higher out-of-pocket (OOP) costs, those who were hospitalized, and those who received Medicare low-income subsidy.

Investigators, led by Ami Vyas, Ph.D., aimed to identify the predictor of adherence to oral cancer medicines. They conducted a retrospective cohort study using data from Optum Clinformatics Data Mart claims database. Patients with a new pharmacy claim for an oral cancer medicine between July 1, 2010, and Dec. 31, 2017, were included, and adherence was assessed after six months of treatment. Vyas is assistant professor of pharmacy practice, specialized in the area of health outcomes research, at the College of Pharmacy, University of Rhode Island, in Kingston, Rhode Island.

Investigators included patients who received oral medications for blood, brain, breast, colorectal, liver, lung, ovarian, prostate, renal, or stomach. They selected these cancers because of their prevalence, higher mortality burden, and indications for oral medications. They did not include older oral endocrine therapies for breast cancer (tamoxifen, anastrozole, and letrozole). They indication that these drugs are generally inexpensive, have been extensively studied, are used to prevent cancer recurrence and metastasis and are not used as front-line cancer therapies.

Their review found that out of 37,938 patients with cancer, 51.9% were adherent to their cancer medication, with adherence ranging from 32.8% among those with liver cancer to 70.4% among those with brain tumor.

The average monthly out-of-pocket costs differed by cancer type, ranging from $749 among patients with blood cancer to $106 among those with prostate cancer. Higher costs were associated with higher odds nonadherence for many cancer types, including renal cancer (and breast cancer. Additionally, patients with inpatient hospitalizations had nonadherence for all cancer types except for stomach cancer.

They also found that the proportion of patients who discontinued medications varied by cancer, with 19.5% of patients with stomach cancer, 30.8% of those with renal cancer, and 49.1% of those with liver cancer who discontinued their cancer meds.

“Our findings suggest that it is crucial to address financial barriers and improve patients’ OAM access to achieve the favorable clinical outcomes demonstrated in clinical trials. Moreover, financial barriers can cause increased emotional distress in addition to the distress already brought on by a facing a life-threatening illness,” investigators wrote.

Of the limitations, investigators said they couldn’t assess adherence by cancer stage because the database used didn’t include cancer staging or dosing differences. Additionally, the database didn’t provide information on patients who may have received copay cards or other assistance or those who were uninsured.

The study was funded by the Pharmacy Quality Alliance and the National Pharmaceutical Council.

Investigators found that socioeconomic and financial barriers can affect patients’ access to oral cancer medications. They suggest that addressing these barriers could improve adherence.

Analysis Confirms High Costs Impact Adherence to Cancer Drugs 

 several drugs from its Medicare formularies, including five that now have generics available and one drug that has been discontinued by the manufacturer.

One of these is the epilepsy drug Vimpat (lacosamide) solution 10 mg/ml from UCB. In March 2022 Alembic, Amneal, and Glenmark launched generic versions of Vimpat. Vimpat has been removed in favor of the generics on some formularies, including Ideal, Value, Value Plus & Essential, and Center of Excellence Formularies. On the Expanded, Complete Formulary, Vimpat will increase in tier. These moves were effective June 8, 2022.

 of Vimpat is $1,098.23 per month, according to UCB. The average retail price of generic lacosamide, 

 to GoodRx, is $878.38 for 60 tablets, but is offered at some locations for as low as $36.22.

Other drugs removed from Prime’s Medicare formularies in favor of generics include:

Takeda’s ulcerative colitis drug Pentasa (mesalamine) 500 mg extended release capsules has been removed from the following formularies: Ideal, Value, Value Plus & Essential, and Center of Excellence Formularies effective June 9, 2022. Sun Pharmaceutical’s generic mesalamine was approved in May 2022. The retail price of 240 capsules of Pentasa is about $1,442.47, and the average retail price of the generic is $473.86 with prices as low as $107.02, 

Lilly’s chemotherapy Alimta (pemetrexed) for IV solution has been removed from Prime’s Ideal, Value, Value Plus & Essential, and Center of Excellence Formularies because of the availability of generics effective June 22, 2022. It is used to treat patients with non-small cell lung cancer and mesothelioma. The list price of Alimta is $10,781.33 per month, according to 

AbbVie’s antidepressant Viibryd (vilazodone) tablets has been removed from Prime’s Ideal, Value, Value Plus & Essential, and Center of Excellence Formularies effective June 23, 2022. Generics of vilazodone are offered by Accord Healthcare, Alembic Pharmaceuticals, Apotex and InvaGen Pharmaceuticals. It is available in 10 mg, 20 mg and 40 mg tablets. Generics of Viibryd have an average retail price of $288.61 and are offered as low a $43.73 for 30 tablets, 

Ortho Dermatologics’ Targretin (bexarotene) gel 1% has been removed from Prime’s Ideal, Value, Value Plus & Essential, and Center of Excellence Formularies because of the availability of generics effective June 28, 2022. It is used to treat patients with cutaneous lesions in patients with cutaneous T-cell lymphoma.

Additionally, TG Therapeutics’ Ukoniq (umbralisib) has been removed from Prime’s formularies because the drug has been discontinued. 

Related: FDA Withdraws Approval of Ukoniq

 its approval for the cancer medicine Ukoniq, which was approved to treat two specific types of lymphoma: marginal zone lymphoma and follicular lymphoma. Updated findings from the UNITY-CLL clinical trial continued to show a possible increased risk of death in patients receiving Ukoniq for the treatment of patients with chronic lymphocytic leukemia. As a result, regulators determined the risks of treatment with Ukoniq outweigh its benefits.

Prime has removed six drugs from its formularies because generics are now available. One has been removed because it has been discontinued.

Prime Therapeutics Updates Medicare Formularies

 Stelara (ustekinumab) to treat children six years of age and older with active psoriatic arthritis (PsA). Stelara is a fully human monoclonal antibody that selectively inhibits both interleukin (IL)-12 and IL-23, two cytokines thought to play an important role in inflammatory response in several autoimmune diseases.

The Janssen Pharmaceutical Companies of Johnson & Johnson 

 a supplemental biologics license application to the FDA in October 2021. The submission is based on data extrapolated from a total of nine studies in adult trials in active psoriatic arthritis and adult and pediatric studies in moderate-to-severe plaque psoriasis.

“We know active pediatric psoriatic arthritis is a challenging inflammatory disease given its rarity and that symptoms, such as swollen joints and skin lesions, can vary significantly in presentation and severity,” Terence Rooney, M.D., Ph.D., vice president, rheumatology and maternal fetal disease area, Janssen Research & Development, said in a press release.

With the limited availability of children with psoriatic arthritis for inclusion in clinical trials, researchers used an extrapolation approach based on previous pharmacokinetic, efficacy and safety observations from a population of pediatric patients with moderate-to-severe plaque psoriasis who also had active psoriatic arthritis, as well as adult patients with moderate-to-severe plaque psoriasis or psoriatic arthritis.

An analysis of the data demonstrated that pharmacokinetic exposure of Stelara in these pediatric patients was consistent with that of phase 3 clinical trials in pediatric patients with plaque psoriasis who did not have active psoriatic arthritis, as well as with adult patients. Data on common efficacy endpoints were similar in these pediatric patients.

Stelara is approved in the United States for the treatment of: adults and children 6 years and older with moderate-to-severe plaque psoriasis; adult patients with active psoriatic arthritis; adult patients with moderately to severely active Crohn’s disease or ulcerative colitis.

Juvenile psoriatic arthritis accounts for about 5% to 8% of all cases of juvenile arthritis cases. It is characterized by both joint inflammation and psoriatic skin lesions that resemble adult psoriatic arthritis.

Stelara is the first biologic targeting both cytokines IL-12 and IL-23 and offers a therapeutic option for children six years of age and older living with active psoriatic arthritis, a rare disease.




FDA Approves Stelara in Children with Psoriatic Arthritis

Optum, a UnitedHealth Group, is working with Sanofi to offer a 30-day supply of some insulins for $35 to people with diabetes who do not have insurance.

“The need for affordable insulin is urgent, especially for uninsured populations,” Heather Cianfrocco, chief executive officer of Optum Rx, said in a 

. “By working with Sanofi, we will improve access and lower costs for people who need this life-saving medication.”

Patients can access a saving card on the 

, which they can take to their pharmacy to receive the $35 price. Insulins that are part of the program include:

• Admelog (insulin lispro injection) 100 Units/mL

• Toujeo (insulin glargine injection) 300 Units/mL

• Toujeo Max (insulin glargine injection) 300 Units/mL

• Lantus (insulin glargine injection) 100 Units/mL

• Apidra (insulin glulisine injection) 100 units/mL

These are the same insulins that Sanofi is offering for uninsured patients through its Insulins 

 Savings Program. Effective July 1, 2022, Sanofi lowered the out-of-pocket cost of these insulins to $35 for a 30-day supply.

“This collaboration supports Sanofi’s ambition to ensure every person in the U.S. living with diabetes can access and afford the insulin they need,” Adam Gluck, senior vice president of U.S. corporate affairs, Sanofi, said in a press release. “This collaboration builds on the many programs, like the Insulins Valyou Savings Program, we’ve implemented to help to deliver on our goal to reduce patient out-of-pocket costs by working with Optum to create yet another access point for affordable insulin.”

For patients with insurance, UnitedHealthcare announced in July 2022 that it will eliminate out-of-pocket costs for some preferred prescription drugs, including some insulins. Beginning in January 2023, patients in standard fully-insured group plans will now have a $0 copay for some short and long-acting insulins, as well as several drugs used to treat emergencies such as severe allergic reactions, hypoglycemia, opioid overdoses, and acute asthma attacks.

Related: UnitedHealthcare to End Copays for Some Drugs

Optum has teamed up with Sanofi to provide savings cards for six insulins through the Optum Store.

Optum to Offer $35 Insulin to Those without Insurance

The FDA has accepted for Fresenius Kabi biologics license application (BLA) for a biosimilar of Genentech’s Actemra (tocilizumab), a monoclonal antibody that is used to treat patients with moderate-to-severe rheumatoid arthritis. Additionally, the FDA has issued an emergence use authorization for Actemra to treat patients who are hospitalized with COVID-19.

Fresenius Kabi expects to receive FDA approval for its tocilizumab biosimilar in 2023 and is seeking approval for both intravenous and subcutaneous routes of administration.

The submission includes analytical similarity and comprehensive clinical data. These are used to demonstrate similar pharmacokinetic, efficacy, safety, tolerability, and immunogenicity to the reference product, with and without switch from Actemra patients to the biosimilar. The BLA includes presentations for both subcutaneous (prefilled syringe and autoinjector) and intravenous administrations.

“The FDA’s acceptance of the BLA for our proposed tocilizumab biosimilar candidate is an important step forward in bringing a wider range of affordable and accessible autoimmune treatment options to patients and healthcare providers while reducing the cost burden to healthcare systems,” Dr. Michael Schönhofen, Fresenius Kabi chief operating officer, said in a press release.

Genentech’s Actemra is under review by the FDA for full approval for treatment of COVID-19 in hospitalized adults who are receiving systemic corticosteroids and require supplemental oxygen. During the COVID-19 pandemic, Genentech struggled with supply shortages, but as of July 2022, the company 

Fresenius Kabi expects to receive FDA approval for its tocilizumab biosimilar in 2023 and is seeking approval in both intravenous and subcutaneous  administration.

FDA Accepts BLA for Biosimilar of Actemra

 development of a therapy to treat dilated cardiomyopathy, a disease in which the heart doesn’t pump efficiently. The phase 3 trial, REALM-DCM, was evaluating PF-07265803 in patients with symptomatic dilated cardiomyopathy (DCM) as the result of a mutation of the gene encoding the lamin A/C protein (LMNA). Results indicated the trial is unlikely to meet its primary endpoint, the change from baseline in the six-minute walk test at 24 weeks.

“This development confirms the complexity of advancing new treatments for rare cardiovascular diseases and the need to further increase knowledge in this space. We thank the patients, families, investigators and members of the advocacy community who contributed to this research,” Chris Boshoff, M.D., Ph.D., chief development officer, oncology and rare disease, Pfizer Global Product Development, said in a press release. “Although this outcome is disappointing, Pfizer remains committed to continuing our work to evolve the treatment paradigm for patients with rare cardiovascular diseases.”

The therapy (formerly ARRY-371797) was obtained by Pfizer as part of its 2019 acquisition of Array Biopharma. It is a small-molecule inhibitor of the p38α mitogen activated protein kinase pathway, which demonstrated improvement in functional capacity in a phase 2 study.

Dilated cardiomyopathy is a disease that often starts in the heart’s left ventricle, and the heart muscle dilates, stretches and becomes thinner. As the heart becomes weaker, heart failure can occur. Dilated cardiomyopathy is the third leading cause of heart failure in the United States. There is no specific treatment for DCM as a result of this gene mutation.

Results indicated the trial is unlikely to meet its primary endpoint, the change from baseline in the six-minute walk test at 24 weeks.  

Pfizer Discontinues Late-Stage Cardiomyopathy Trial

The Centers for Disease Control and Prevention has added a diagnosis code to the International Classification of Diseases, 10th Revision, Clinical Modification (ICD-10-CM) for reporting cases of activated phosphoinositide 3-kinase delta syndrome (APDS). The diagnosis code, D81.82, will be effective starting Oct., 1, 2022, and will allow the CDC and physicians to identify and report on this rare primary immunodeficiency.

“By assigning this ICD-10-CM code, the CDC is formally recognizing APDS as a discrete immunological disease, and that will make a life-altering difference for people affected by the condition. By using the unique diagnostic code to identify both established and new patients with APDS, physicians will increase care options for affected individuals while helping to boost the world’s understanding of the prevalence, mechanisms, and outcomes of this progressively debilitating disease,” Anurag Relan, M.D., chief medical officer of Pharming, said in a 

APDS is a rare primary immunodeficiency that affects about one to two people per million. Also known as PASLI, it is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Diagnosis is often difficult because of the wide variety of symptoms patients experience.

Pharming is developing a therapeutic, leniolisib, to treat patients 12 years of age and older who have activated PI3K delta syndrome.

In the United States, Pharming filed a new drug application in July 2022 with leniolisib for the treatment of APDS in adults and adolescents aged 12 or older. In Europe, the European Medicines Agency’s Committee for Medicinal Products for Human Use granted an accelerated assessment for Pharming’s application for leniolisib. Pharming plans to submit its marketing authorization application for leniolisib to the EMA in October 2022.

The applications include positive data from a phase 2/3 study, which met both its co-primary endpoints in the target patient population of evaluated reduction in lymph node size and correction of immunodeficiency.

 that the multinational, phase 3 portion of the clinical trial, conducted by Novartis, met its co-primary endpoints, which evaluated reduction in lymph node size and correction of immunodeficiency. The shrinking of lymphadenopathy lesions and increased proportion of naïve B cells are important in this population, as they indicate a reduction in APDS disease markers.

The new code, effective Oct. 1, 2022, allows APDS to be recognized as an distinct immunological disease.

CDC Adds Diagnosis Code for Rare Primary Immunodeficiency 

The phase 3 LEAP-002 trial investigating Merck’s Keytruda (pembrolizumab) plus Eisai’s Lenvima (lenvatinib) 

 its endpoints of overall survival and progression-free survival as a first-line treatment for patients with unresectable hepatocellular carcinoma. The combination compared with Lenvima alone trended toward improvement in overall survival and progression-free survival for patients but did not meet statistical significance.

This is the second Keytruda trial failure that Merck has announced in less than a month. In July, Keytruda failed to meet the primary endpoint of event-free survival for the treatment of patients with unresected locally advanced head and neck squamous cell carcinoma (HNSCC). The phase 3 KEYNOTE-412 trial was evaluating Keytruda with concurrent chemoradiation therapy followed by Keytruda as maintenance therapy.

Related: Keytruda Fails in Phase 3 Trial for Head and Neck Cancer

In the LEAP-002 study, the median overall survival of the Lenvima monotherapy arm was longer than that observed in previously reported clinical trials evaluating this therapy as a monotherapy in unresectable hepatocellular carcinoma. The safety profile of Keytruda/Lenvima was consistent with previously reported data on the combination.

“Our joint clinical development program for Keytruda plus Lenvima is designed to address unmet needs for some of the most challenging-to-treat types of cancer, like hepatocellular carcinoma,” Gregory Lubiniecki, M.D., vice president, global clinical development, Merck Research Laboratorie, said in a press release. “We remain confident in the potential of this combination based on the body of evidence we’ve seen to date and will continue to investigate its role across multiple types of cancer.”

In the United States, the Keytruda/Lenvima combination is approved to treat patients with advanced endometrial carcinoma who have disease progression and for adult patients with advanced renal cell carcinoma in the first-line setting.

Hepatocellular carcinoma is the most common type of primary liver cancer and the most rapidly increasing cause of cancer deaths in the United States. Hepatocellular carcinoma accounts for approximately 90% of primary liver cancers. It is estimated there were more than 905,000 new cases of liver cancer and more than 830,000 deaths from the disease globally in 2020.

Merck and Eisai are also studying the Keytruda/Lenvima in more than 10 different tumor types — endometrial carcinoma, hepatocellular carcinoma, melanoma, non-small cell lung cancer, renal cell carcinoma, head and neck cancer, biliary tract cancer, colorectal cancer, gastric cancer, esophageal cancer, glioblastoma and pancreatic cancer — across more than 15 clinical trials.

This is the second trial in less than a month studying Keytruda that failed to meet endpoints.

Keytruda/Lenvima Combo Fails in Liver Cancer Trial

Two specialty medications to treat rare diseases, along with novel treatments for myeloma and plaque psoriasis, are highlighted in OptumRx’s new Drug Pipeline Insights report. The top four medications —teclistamab, deucravacitinib, betibeglogene autotemcel, and elivaldogene autotemcel— are expected to receive FDA approval in the third quarter.

 (brand name to be determined) is a novel oral drug being evaluated to treat adults with moderate-to-severe plaque psoriasis developed by Bristol Myers Squibb. Plaque psoriasis affects about 6 million people in the United States. Deucravacitinib is a novel tyrosine kinase 2 (TYK2) inhibitor that regulates overproduction of immune inflammatory compounds in immune-mediated diseases such as psoriasis, OptumRx said.

While deucravacitinib would provide a first-in-class oral treatment for a condition with limited oral options, it is still entering a crowded marketplace, according to OptumRx.

Injectable biologics are commonly used with high response rates in patients with moderate-to-severe plaque psoriasis. Immunosuppressants such as methotrexate and cyclosporine have historically been used, but their place in therapy is limited because of safety concerns, according to the PBM.

Celgene’s Otezla (apremilast), an oral phosphodiesterase 4 (PDE4) inhibitor, has been available since 2014 and has a safer adverse event profile versus historical oral treatments but its use has been limited because injectable biologics are more effective, OptumRx said. In trials, deucravacitinib demonstrated superior efficacy versus Otezla and was associated with numerically lower rates of discontinuations due to adverse events.

“Deucravacitinib is indeed entering a crowded marketplace with multiple treatments already available,” Bill Dreitlein, Pharm.D., senior director, pipeline and drug surveillance at OptumRx, told 

. “However, psoriasis is an $11 billion marketplace and deucravacitinib will aim to carve out a slice of that as a new oral option.”

An FDA decision is expected Aug. 19, 2022.

(brand name to be determined), administered subcutaneously, is a novel drug to treat relapsed or refractory multiple myeloma developed by Janssen. Teclistamab is a bispecific antibody that targets both B-cell maturation antigen (BCMA) and CD3, a T-cell receptor.

It enters a competitive field, with three similar therapies that target BCMA for relapsed or refractory multiple myeloma, OptumRx said. Existing medications include:GSK’s Blenrep(belantamab mafodotin), which is administered intravenously; along with CAR-T cell therapies Carvykti (ciltacabtagene autoleucel) from Janssen Biotech and Abecma (idecabtagene vicleucel) from Bristol Myers Squibb.

Indirectly, teclistamab appears more effective than Blenrep, but less effective versus the CAR-T cell therapies, according to OptumRx. “However, it appears that the CAR-T cell therapies see higher rates of CRS and neurotoxicity than teclistamab,” the PBM said.

A wholesale acquisition cost for teclistamab has not been announced, according to OptumRx.

An FDA decision is expected Sept.10, 2022.

, with a brand name of Zynteglo, is a gene therapy for transfusion-dependent beta-thalassemia developed by Bluebird Bio. If approved, Zynteglo could be a cure for the rare disease through a one-time intravenous infusion, according to OptumRx.

Beta-thalassemia is caused by genetic mutations in cells that carry oxygen throughout the body, which may produce classic signs of anemia and may be life-threatening if left untreated, according to OptumRx.

Current treatment for severe beta-thalassemia is mainly regular, life-long red blood cell transfusions. Patients also need iron chelation therapy to address excess levels of iron in the body due to the repeated blood transfusions.

Stem cell transplantation from a matched donor, ideally a sibling, is currently the only possible cure. However, stem cell transplants carry their own risks, and fewer than 25% of patients have access to a suitable match, Dreitlein said.

The only other FDA-approved therapy for treatment transfusion dependent beta-thalassemia is Reblozyl (luspatercept) developed by Bristol Myers Squibb and Acceleron, which only treats the anemia resulting from the disease and requires an injection every three weeks.

“If approved, Zynteglo would be a one-time treatment and the first gene therapy for a blood disorder,” Dreitlein said. As a result, it could “break the ice” for other treatments in the future for other blood disorders such as sickle cell disease, he added.

Of the estimated 3,000 patients with beta-thalassemia in the United States, about half of them are dependent on blood transfusions. “The target population for this treatment is extremely small. Bluebird bio estimates about 1,500 patients with beta-thalassemia in the United States would be eligible,” OptumRx said.

Analysts forecast a $2.1 million cost for a one-time dose of Zynteglo, according to OptumRx.

An FDA decision is expected Aug. 19, 2022

with the brand name Skysona, is a gene therapy to treat cerebral adrenoleukodystrophy (CALD) in young boys developed by Bluebird Bio. Patients with CALD lack a protein (ALDP) needed to break down fatty acids, which causes toxic acids to build up in the brain and leads to disability and an early death. Most patients with CALD will die within a decade of diagnosis if they are not treated with bone marrow (stem cell) transplantation.

There are around 40 patients with CALD in the United States annually, according to OptumRx.

“If approved, Skysona would be a one-time treatment that may stabilize disease progression and preserve neurological function in patients with early CALD,” OptumRx said.

While Bluebird bio has not announced a price for Skysona, the wholesale acquisition cost for Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for spinal muscular atrophy, is $2.125 million, according to Optum Rx.

An FDA decision is expected Sept. 16, 2022.

OptumRx profiles four therapies that are expecting FDA decisions in the next few months.

FDA Accepts BLA for Biosimilar of Actemra

Pfizer Discontinues Late-Stage Cardiomyopathy Trial

CDC Adds Diagnosis Code for Rare Primary Immunodeficiency

Keytruda/Lenvima Combo Fails in Liver Cancer Trial

Top 4 Drugs to Watch in the Third Quarter