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FDA Approves Tx for Ultra-Rare ALS Condition – Qalsody

This week the FDA approved a new therapy, Qalsody (tofersen) from Biogen MA, Inc., for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. Continued approval for this indication is contingent upon providing more data on the drug’s effectiveness and clinical benefit in confirmatory, post-launch trial(s).

Qalsody is the first available treatment targeting a genetic cause of the disease. Approval is based on trial data that showed it reduced levels of neurofilament protein, an indicator of nerve cell degeneration believed to be tied with disease progression. 

According to the CDC, between 16,000 and 32,000 Americans are currently living with ALS. Approximately 2% of ALS cases are associated with mutations in the SOD1 gene. The agency estimates there are fewer than 500 patients with SOD1-ALS in the United States.

Qalsody is administered by intrathecal injection by a trained health care provider. Patients receive three initial doses administered at 14-day intervals, followed by a maintenance dose every 28 days.

Biogen confirmed that it expects to launch Qalsody on par with other ALS therapies on the market. Relyvrio, approved for ALS in 2022, costs about $158,000 annually, while older Radicava costs about $170,000 annually.

Qalsody is likely to launch through specialty pharmacy distribution given its cost, very small patient population and requirements to capture real-time clinical data.

CLICK HERE for prescribing information


FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene

04/25/2023 — FDA approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Qalsody is an antisense oligonucleotide that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. The approval was based on a reduction in plasma neurofilament light (NfL), a blood-based biomarker of axonal (nerve) injury and neurodegeneration. 

Disease or Condition

Similar to sporadic ALS, where there are no associated risk factors and no family history of the disease, SOD1-ALS is a progressive neurodegenerative disease that attacks and kills the nerve cells that control voluntary muscles. Voluntary muscles produce movements such as chewing, walking, breathing, and talking. ALS causes the nerves to lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis.


The effectiveness of Qalsody was evaluated in a 28-week, randomized, double-blind, placebo-controlled clinical study in 147 patients with weakness attributable to ALS and a SOD-1 mutation confirmed by a central laboratory. The study randomly assigned 108 patients in a 2:1 ratio to receive treatment with either Qalsody 100 mg (n = 72) or placebo (n = 36) for 24 weeks (three loading doses followed by five maintenance doses).

The participants were approximately 43% female; 57% male; 64% White; and 8% Asian. The average age was 49.8 years (range from 23 to 78 years).

Patients receiving Qalsody had nominally significant reductions in plasma NfL concentration at Week 28 compared to the placebo arm. The findings are reasonably likely to predict a clinical benefit in patients. The observed reduction in NfL was consistent across all subgroups based on sex, disease duration since symptom onset, site of onset, and use of other medications for ALS treatment.

Qalsody is approved under the accelerated approval pathway, under which FDA may approve drugs for serious conditions where there is an unmet medical need and a drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. To confirm the clinical benefit of Qalsody, a Phase 3 randomized, double-blind, placebo-controlled trial is ongoing in individuals who are carriers of the SOD1 genetic mutation who do not yet have symptoms. The study will assess the proportion of individuals treated with Qalsody who develop symptoms of ALS during the trial compared to placebo. 

Safety Information

The most common side effects were pain, fatigue, arthralgia (joint pain), increased cerebrospinal (brain and spinal cord) fluid white blood cells, and myalgia (muscle pain).


Qalsody received priority review, orphan drug and fast track drug designations.


Pharmacy Sued for Switching Generic to Generic

Most would agree that the US has been a litigious-heavy country for decades. As we know, healthcare is one of the biggest targets for lawsuits of every complexion. And under the healthcare umbrella, pharmacy follows physicians and hospitals as a major target for litigation.

We’ve seen lots of lawsuits filed by patients claiming harm following a refill with a generic drug switched at the pharmacy from their beloved brand name product. However, the article below details the case of a patient claiming harm when their generic drug was dispensed by the same generic drug….. but from a different manufacturer. Who’d a expect that?

One reason that we are covering this lawsuit in today’s Report is that the pharmacist and even the pharmacy technician were named in the suit. The pharmacist and technician were eventually released from the suit but experienced the hassle and expense of mounting a defense. Also not good PR in the media.

Specialty pharmacies should always be on the watch for potential litigation. The case below is a learning experience for SP management and should be shared with pharmacy legal counsel.


Patient Files Negligence Suit After Receiving a Different Generic Version

Apr 24, 2023

Individual alleges that an equivalent product from another manufacturer precipitated series of seizures.

Issue of the Case

A patient who presented a prescription for dispensing received an equivalent product from a different manufacturer and alleged that a series of seizures resulted from the change. Will she prevail in her negligence lawsuit against the pharmacy?

Facts of the Case

The patient had been taking the antiseizure medication levetiracetam for 10 years, always receiving it in a form described as yellowish white. She presented a new prescription at a chain pharmacy and received a pink version of the medication. She asked the pharmacist whether this new version would be safe to use; he told her it would.

After starting to use the new version, the patient experienced 3 seizures, which led to her being admitted to the hospital. This was cause for concern, because she had been using the yellowish-white version for 10 years and had not had a seizure for 7 years.

She filed a lawsuit against the pharmacy, the pharmacist, and the pharmacy technician involved in dispensing the medication. The patient subsequently added the manufacturer of the pink dosage form as a defendant.

The patient’s attorney advanced several legal theories as the basis for liability and made submissions to the state trial court that had jurisdiction. A total of 4 amended complaints were submitted on behalf of the patient as the matter progressed. As is sometimes appropriate, a complaint would be submitted and rejected by the court but with authorization to revise it and resubmit. Through this process, the pharmacist and technician were deleted as defendants, but the matter was decided as amended against the pharmacy chain and the manufacturer.

The trial court judge received a motion from the attorneys for the defendants to dismiss the lawsuit and reviewed the legal arguments and theories advanced by the attorneys for the patient. All arguments were rejected and the lawsuit was dismissed. The plaintiff appealed to the state’s appellate court.

The Ruling

The appellate court reviewed the various arguments of the plaintiff and the lower court judge’s decisions. All decisions were affirmed on appeal. The matter was dismissed.

The Court’s Reasoning

The negligence claim advanced by the plaintiff argued that the pharmacy had a legal duty to dispense the medication without a change in source, as well as to warn of potential dangers associated with the version dispensed. The pharmacy chain responded that the prescription was issued using generic product identification and that the various expectations found in the state’s drug product selection statute, which were the basis for the plaintiff’s arguments, did not apply. Moreover, the chain argued that any duty to warn of danger associated with the medication rested with the prescriber under the learned intermediary doctrine. The appellate court agreed with the pharmacy chain.

Next, the patient’s allegation that the pharmacist’s response that use of the different version of medication would be OK did not give rise to any legal duty on the part of the pharmacist or pharmacy in the view of the appellate court judges.

The plaintiff’s attorneys also argued that the pharmacy had a legal duty to dispense the medication without changing the source. The court agreed with the pharmacy chain that because the prescriber had not marked the portion of the prescription form that read “DAW [dispense as written]: No product selection indicated,” that interchange of product source had not been prohibited by the prescriber.

In another revision, the plaintiff argued that the sub potency of the dispensed product resulted in it being considered adulterated. That led to the argument that the pharmacy had been negligent because of obtaining tablets in an adulterated state that were initially made in a form less than 500 mg. The pharmacy chain responded that it was not the manufacturer of the dosage forms and thus the plaintiff should not prevail with this argument. The court agreed.

The manufacturer of the tablets that had been dispensed argued that the FDA had found its version to be therapeutically equivalent to the product the patient had originally received. If there were a difference in therapeutic effect, the sole enforcement authority for that rested with the FDA under a specific provision in the relevant federal statute. The trial court judge dismissed that argument by the plaintiff, and the court of appeals affirmed it.

Finally, the plaintiff had argued that the pharmacy knew about the purported defect in the medication dispensed and had ignored that, resulting in her injury. In response, the defendants pointed out that plaintiff had been in possession of the allegedly subpotent dosage forms for more than 2 years with an opportunity to examine the chemical composition of the tablets. The plaintiff had never allegedly pursued such testing and had made no direct allegation of a defect existing in the dosage forms.

In conclusion, the court of appeals rejected all arguments advanced by the plaintiff in the various amended complaints, affirmed the decisions of the trial court, and, overall, affirmed dismissal of the lawsuit.

About the Author

Joseph L. Fink III, JD, DSS (Hon), BSPharm, FAPHA, is professor emeritus of pharmacy law and policy at the U of Kentucky College of Pharmacy in Lexington.

Pharmacy Times


Is a Payer Not a Purchaser? Court Says No

The article below is one that a specialty pharmacy’s legal counsel might find most interesting. In short, the US District Court ruling below said that Humana could not pursue RICO charges against Biogen and ACS Pharmacy because it did not have standing in the matter. Humana would have needed to be the “purchaser” to have standing and the court deemed it was only the “payer”.  Hmmmm

The difference between purchaser vs. payer may be difficult for the non-legal-eagles in our midst to fully fathom. But it does clarify how the courts perceive the manufacturer and the specialty pharmacy in these instances. And, ultimately, specialty pharmacies can face a much bigger legal hill to climb if charged with a RICO violation.

Fortunately for the industry, there have been fewer legal black-eyes related to financial shenanigans by specialty pharmacies in recent years. That’s good for the industry especially in a reactionary legislative environment.

Click here to read the full text of the opinion.


RICO – Standing – Indirect Purchaser

U.S. District Court

By: Mass. Lawyers Weekly Staff April 6, 2023

Where a plaintiff insurance company has filed a complaint under the Racketeer Influenced and Corrupt Organizations statute alleging that the defendant made unlawful charitable donations to fund patient copays of its multiple sclerosis drugs, the defendant’s motion to dismiss should be allowed because the plaintiff is an indirect purchaser of the pharmaceutical products at issue, so it does not have standing to assert a civil RICO claim.

“This is an action arising out of an alleged scheme to increase the number of prescriptions of drugs used to treat multiple sclerosis (‘MS’) through improper charitable contributions. Plaintiff Humana, Inc. is a health-insurance company. Defendant Biogen, Inc. is a biotechnology company and manufacturer of three different drugs used to treat MS. Defendant Advanced Care Scripts, Inc. (‘ACS’) is a specialty pharmacy company. According to the complaint, Biogen made unlawful donations to different charities to fund patient copays of its MS drugs, thereby increasing the sales of those drugs.

“… Although Humana was the ultimate payor of the prescription drugs at issue — putting to one side the requirement of a patient copay — it did not purchase the drugs directly from Biogen. Instead, it paid pharmacies for the drugs, who had purchased the drugs from wholesalers or distributors (or possibly from Biogen itself). Humana was thus an ‘indirect’ purchaser.

“Under the ‘indirect purchaser rule,’ first developed by the Supreme Court in the antitrust context, only a direct purchaser of goods has standing to assert a claim for violation of the antitrust laws. Every circuit to have considered the issue has held that the rule also applies to civil RICO actions, and that indirect purchasers therefore do not have standing to assert RICO claims. The First Circuit has not yet addressed the question. While there may be practical and policy reasons to question the application of that rule in the health-insurance context, for the reasons that follow, this Court will follow the majority rule. It will therefore dismiss the civil claims for failure to state a claim on that basis. …

“Accordingly, because Humana is an indirect purchaser of the pharmaceutical products at issue in this case, it does not have standing to assert a civil RICO claim. Count 1 will therefore be dismissed.”

Humana, Inc. v. Biogen, Inc., et al. (Lawyers Weekly No. 02-154-23) (34 pages) (Saylor, C.J.) (Civil Action No. 21-11578-FDS) (March 31, 2023).


FDA Approves New Tx for Rare Eye Condition – Syfovre

The FDA recently approved a therapy previously approved in 2021….. but with a very different indication and a new NDC. The approval was granted for Syfovre (pegcetacoplan injection) from Apellis Pharmaceuticals, Inc for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). It is the first and only therapy approved to treat geographic atrophy.  It is approved for intravitreal administration only by a qualified physician.

This is the second approval for the drug. The FDA first approved pegcetacoplan in paroxysmal nocturnal hemoglobinuria, a rare blood disorder where the immune system attacks red blood cells and platelets. It is marketed as Empaveli in that indication.

Although the approval did not include a black box warning the therapy can trigger ocular and periocular infections, active intraocular inflammation, endophthalmitis and retinal detachments among other adverse events.

The recommended dose for Syfovre is 15 mg (0.1 mL of 150 mg/mL solution) administered by intravitreal injection to each affected eye once every 25 to 60 days.

The injection should be on the market by the beginning of March. The company announced launch price at $2,190 per vial before discounts. That translates into between $13,200 and $26,400 annually based on 30 vs. 60-day dosing.

Apellis did not announce distribution plans for Syfovre. Given its cost it is likely that it will be distributed by a specialty pharmacy distributor.

CLICK HERE to access prescribing information


US FDA approves Apellis’ geographic atrophy therapy Syfovre

Syfovre is indicated for geographic atrophy patients with or without subfoveal involvement.

The US Food and Drug Administration (FDA) has granted approval for Apellis Pharmaceuticals’ Syfovre (pegcetacoplan injection) to treat geographic atrophy (GA), an advanced form of age-related macular degeneration (AMD).

Syfovre has been approved for use in people with GA with or without subfoveal involvement and offers to dose flexibility with a regimen of every 25 to 60 days for patients and physicians.  It provides comprehensive control of the complement cascade, which is part of the immune system of the body, by targeting C3.

Apellis Pharmaceuticals co-founder and CEO Cedric Francois said: “Today marks an extraordinary milestone for patients, the retina community, and Apellis. With its increasing effects over time and flexible dosing, we believe that Syfovre will make a meaningful difference in the lives of people with GA. GA is a complex disease that the field has spent decades trying to address, so we are humbled and proud to bring forward the first-ever treatment.”

The regulatory approval is based on positive data from the Phase III OAKS and DERBY trials conducted across a broad and representative population of GA patients. Syfovre reduced the GA lesion growth rate compared to sham injections in the OAKS and DERBY studies.

It also showed increasing effects of treatment over time, with up to 36% lesion growth reduction occurring between months 18 and 24. Syfovre’s safety profile is well-demonstrated after approximately 12,000 injections over 24 months.


Pharma CEOs Agree Like Never Before

Ever get a letter from more than 400 people? 

Not 400 letters…. 1 letter with four hundred+ signatories!

Over the last week more than 400 of the tippity topest brass in Pharma signed onto an open letter protesting the recent Texas court ruling blocking the sales of Mifepristone. Pharma’s response is generating as much news as the original ruling.

So what’s the unique motivation for so many big Pharma heavy weights to raise the alarm? Money and market stability

The primary worry is that a court could, on a whim (or a bias), rule that ANY drug approved by the FDA should be delisted nationally.  A monkey wrench in the gears could cost a manufacturer a huge amount of money even in the short term due to loss of revenue while sidelined, the disruption of physician prescribing preference, and the loss of patient confidence in their therapy….. just to cite a few impacts.

Expand the theory a bit and the same ‘legal logic’ could be applied to drugs in development questioning validity of drug trials and the FDA review and approval process. Drug development is very expensive and ‘time to launch’ in a competitive market translates into BIG money. Even the specter of such events would make any Pharma CEO quake in their lab booties.

CLICK HERE to read the open letter….. and even add your signature while at it.


300 Biotech and Pharma CEOs Call for a Reversal of the Abortion Pill Ban

An open letter calls for a potential ban on abortion pill mifepristone to be reversed.

April 10, 2023 — Conflicting court rulings by federal judges in Texas and Washington last week left the future of a commonly used abortion pill in doubt. Now, more than 300 executives from biotech and pharmaceutical companies have signed an open letter opposing a Texas judge’s decision to stop the pill from being sold.

“We call for the reversal of this decision to disregard science, and the appropriate restitution of the mandate for the safety and efficacy of medicines for all with the FDA, the agency entrusted to do so in the first place,” the CEOs wrote in the letter.

The CEO of pharma giant Pfizer, Albert Bourla, signed the letter, along with hundreds of smaller American biotech companies, including ReCode Therapeutics, Blackfynn, and Ovid Therapeutics. 

Roe v. Wade was overturned by the U.S. Supreme Court last summer, which had previously granted access to abortion health care nationwide. In the aftermath of that reversal, states independently decided whether or not abortion would be allowed or effectively banned. 

Last Friday, Trump-appointed U.S. District Judge Matthew Kacsmaryk ordered a suspension of the federally approved mifepristone, a drug that has been used for over two decades to terminate pregnancies. On the same day, Washington State’s U.S. District Judge Thomas O. Rice, who was appointed by the Obama administration, called for the opposite and directed the FDA not to roll back the abortion pill. 

“If courts can overturn drug approvals without regard for science or evidence, or for the complexity required to fully vet the safety and efficacy of new drugs, any medicine is at risk for the same outcome as mifepristone,” the CEOs’ letter says.

By Prarthana Prakash, Fortune Magazine


Limited Distribution Deals Announced

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 three LD deals that have been recently publicly confirmed subsequent to the approvals.

ORSERDU Available through Biologics by McKesson

CARY, N.C., Feb. 10, 2023—Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, was selected by Stemline Therapeutics Inc., as a limited distribution network specialty pharmacy provider for Orserdu (elacestrant).

ORSERDU Available through Onco360

ORSERDU is an estrogen receptor antagonist indicated for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy.

Biologics Named Exclusive Pharmacy for SKYCLARYS (omaveloxolone)

Biologics by McKesson was selected by Reata Pharmaceuticals as the sole specialty pharmacy provider for Skyclarys (omaveloxolone), the first and only treatment for Friedreich’s ataxia (FA). Skyclarys was approved by the U.S. Food and Drug Administration (FDA) on Feb. 28, 2023, for the treatment of FA in adults and adolescents aged 16 years and older, along with a rare pediatric disease priority review voucher.


The Holy Grail of Specialty – Limited Distribution

If you have you ever tried to explain all the nuances behind the host of decisions to launch a new specialty therapy through limited distribution, then look no further. The article below does a yeoman’s job of explaining the ins and outs, ups and downs, and even some sideways of LD.

Limited distribution is the holy grail for a specialty pharmacy. Being selected as even one of a dozen (or more) specialty pharmacies ensures that the lucky SP will get a slice of what are typically a high ticket (and hopefully high margin $$s) prescriptions. Many a specialty pharmacy has gone from a ‘little engine that could’ to a behemoth SP in short order due to their success in landing limited distribution deals. At the other end of the spectrum, the ‘blessed’ SPs that are selected as the exclusive distribution partner for a new high-ticket therapy can see revenues skyrocket overnight (cash flow is the rocket fuel that SPs need to grow). 

SPs learned that breaking the limited distribution glass ceiling was their ticket to the big time. That also means being offered preferred contracts by PBMs and Payers for reimbursement (since the manufacturer’s selection alone doesn’t always guarantee payment.) And, that contract usually opens the door to other non-limited-distribution therapies that might have also been previously blocked to them.

The article below focuses on limited distribution for Oncology products…. which represent the bulk of all LD programs. It also provides some key metrics that determine what qualifies as a limited distribution therapy even beyond Oncology.  New hires working the specialty pharmacy segment would do well to familiarize themselves with this important area of specialty.


Developing a Limited Distribution Plan for Oral Oncology Products

Mar 21, 2023 — The portfolio of oral oncology products has evolved over the past few years and so has the traditional model of distribution.

Distribution strategy is one of several key decisions that a manufacturer must make early in the process when launching a new oral oncology product. As the market has shifted from traditional community-based products to specialty medications, expanded solutions to better serve these products have evolved.

High-touch, oral oncology products require distribution solutions that are targeted toward maximizing patient outcomes. Limited distribution through a carefully selected specialty pharmacy network offers a greater level of patient care but also brings more complexity and the need for greater understanding to identify the right solution.

Why Select a Limited Specialty Pharmacy Distribution Model for Your Product?

CLICK HERE to access the full article

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