US FDA approval and panel tracker: May 2023

 Last month the FDA granted approval for the first and second ever respiratory syncytial virus vaccines, from GSK and Pfizer respectively. Both companies have committed to conducting postmarketing studies to assess signals of Guillain-Barré syndrome and other immune-mediated demyelinating conditions. Another big event for the jabs is looming, with the CDC's advisory committee on immunization practices due to meet on 21 June to discuss how the vaccines should be used. Both companies anticipate launch before the upcoming RSV season. Separately Pfizer also gained a positive adcom for Abrysvo’s use in very young children, via maternal vaccination. Sarepta had its ups and downs in May: after a favourable adcom for its DMD gene therapy contender SRP-9001, the FDA decided to postpone the final decision until 22 June. An accelerated approval is now expected to be restricted to patients aged 4-5 years old. And Intercept's Nash dream came crashing down again as an adcom voted against obeticholic acid, causing shares to sink to a record low. The final nail in the coffin, another CRL, is expected later this month.

This snippet has been updated to include Pfizer's postmarketing plans for its RSV vaccine in older adults.

Notable first-time US approval decisions in May 2023
Project	Company	Indication(s)	2028e SBI ($m)	Outcome
SRP-9001 (delandistrogene moxeparvovec)	Sarepta	Ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene	2,036	Delayed from 29 May until 22 June, positive adcom
Veozah (fezolinetant)	Astellas	Moderate-to-severe vasomotor symptoms associated with menopause	1,904	Approved
Arexvy	GSK	Prevention of lower respiratory tract disease caused by RSV in adults ≥60 years	1,729	Approved
Abrysvo	Pfizer	Prevention of lower respiratory tract disease caused by RSV in adults ≥60 years	1,306*	Approved
Epkinly (epcoritamab, DuoBody-CD3xCD20)	Genmab/Abbvie	Relapsed/refractory large B-cell lymphoma after ≥2 lines of systemic therapy	1,187	Approved (accelerated)
Bimzelx	UCB	Plaque psoriasis	718	FDA issued a Form 438 relating to manufacturing deficiencies
Vyjuvek (B-Vec, beremagene geperpavec)	Krystal	Dystrophic epidermolysis bullosa	636	Approved
Inpefa (sotagliflozin)	Lexicon	Heart failure (HFrEF & HFpEF pts with/out diabetes)	333	Approved
Alhemo (concizumab)	Novo Nordisk	Haemophilia A and B with inhibitors	198	CRL (additional information requested)
Xacduro (sulbactam-durlobactam, Sul-Dur)	Innoviva (Entasis)	Hospital‐acquired and ventilator‐associated bacterial pneumonia caused by Acinetobacter baumannii‐calcoaceticus complex in adults	82	Approved
Mydcombi	Eyenovia	Drug-device combination for in-office pupil dilation (mydriasis)	54	Approved
Elfabrio (pegunigalsidase alfa, PRX-102)	Chiesi/Protalix	Fabry disease	-	Approved
Trastuzumab duocarmazine (SYD985)	Byondis	Her2-positive unresectable breast cancer	-	CRL (additional information requested)
Brixadi	Camurus	Opioid use disorder	-	Approved
Anktiva (N-803)	Immunitybio	BCG-unresponsive non-muscle-invasive bladder cancer	-	CRL (manufacturing deficiencies and CMC problems)
Miebo (NOV03)	Bausch & Lomb	Dry eye disease	-	Approved
Posluma (flotufolastat F 18/ 18F-rhPSMA-7.3)	Blue Earth Diagnostics/ Bracco	PSMA-targeted PET imaging agent for prostate cancer	-	Approved
*Older adults and maternal setting not split out. SBI: sales by indication. Sources: Evaluate Pharma & company releases.

 

Advisory committee meetings in May 2023
Project	Company	Indication	2028e SBI ($m)	Outcome
SRP-9001 (delandistrogene moxeparvovec)	Sarepta	Ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene	2,036	8-6 in favour
Abrysvo	Pfizer	Prevention of (severe) lower respiratory tract disease caused by RSV in infants from birth through 6 months by active immunisation of pregnant individuals	1,306*	In favour, 14-0 on effectiveness and 10-4 on safety
Ocaliva (obeticholic acid)	Intercept	Pre-cirrhotic liver fibrosis due to Nash	663	12-2 against (benefits do not outweigh the risks) 15-1 vote to defer until outcomes data are available
Epinephrine nasal spray (Neffy)	ARS Pharmaceuticals	Emergency treatment of allergic reactions (type I) including anaphylaxis in adults and children ≥30 kg	-	16-6 in favour (adults), 17-5 in favour (children)
Opill	Perrigo	Daily oral contraceptive (progestin-only)	-	17-0 in favour of making Opill available for OTC use
*Older adults and maternal setting not split out. SBI: sales by indication. Sources: FDA adcom calendar, Evaluate Pharma & company releases.

 

Supplementary and other notable approval decisions in May 2023
Product	Company	Indication (clinical trial)	Outcome
Kalydeco	Vertex	Cystic fibrosis in children from 1 month to <4 months of age	Approved
Paxlovid	Pfizer	Patients who are at high risk of progression to severe disease from Covid-19 (additional analyses of Epic-HR and Epic-SR trials)	Approved
Rexulti	Otsuka/ Lundbeck	Agitation associated with Alzheimer’s dementia	Approved
Ayvakit	Blueprint	Indolent systemic mastocytosis (Pioneer)	Approved
Opvee (OPNT003 nasal nalmefene)	Indivior (Opiant)	Opioid overdose (NCT04759768, NCT05219669, NCT04828005)	Approved
Rinvoq	Abbvie	Adult patients with moderately to severely active Crohn's disease (U-Exceed, U-Excel, U-Endure)	Approved
Farxiga	Astrazeneca	Heart failure with preserved ejection fraction (Deliver)	Approved
Yuflyma (high concentration Humira biosimilar)	Celltrion	RA, juvenile idiopathic arthritis, PsA, ankylosing spondylitis, Crohn’s disease, UC, plaque psoriasis and hidradenitis suppurativa	Approved
RA: rheumatoid arthritis; PsA: psoriatic arthritis; UC: ulcerative colitis. Source: Evaluate Pharma & company releases. https://www.evaluate.com/vantage/articles/insights/nme-approvals-snippets/us-fda-approval-and-panel-tracker-may-2023

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Nonprofit Allina Health System Cuts Off Patients With Medical Debt

 Many hospitals in the United States use aggressive tactics to collect medical debt. They flood local courts with collections lawsuits. They garnish patients’ wages. They seize their tax refunds.

But a wealthy nonprofit health system in the Midwest is among those taking things a step further: withholding care from patients who have unpaid medical bills.

Allina Health System, which runs more than 100 hospitals and clinics in Minnesota and Wisconsin and brings in $4 billion a year in revenue, sometimes rejects patients who are deep in debt, according to internal documents and interviews with doctors, nurses and patients.

Although Allina’s hospitals will treat anyone in emergency rooms, other services can be cut off for indebted patients, including children and those with chronic illnesses like diabetes and depression. Patients aren’t allowed back until they pay off their debt entirely.

Nonprofit hospitals like Allina get enormous tax breaks in exchange for providing care for the poorest people in their communities. But a New York Times investigation last year found that over the past several decades, nonprofits have fallen short of their charitable missions, with few consequences.

Allina has an explicit policy for cutting off patients who owe money for services they received at the health system’s 90 clinics. A 12-page document reviewed by The Times instructs Allina’s staff on how to cancel appointments for patients with at least $4,500 of unpaid debt. The policy walks through how to lock their electronic health records so that staffers cannot schedule future appointments.

“These are the poorest patients who have the most severe medical problems,” said Matt Hoffman, an Allina primary care doctor in Vadnais Heights, Minn. “These are the patients that need our care the most.”

Allina Health said it has a robust financial assistance program that in an average year helps over 12,000 of its 1.9 million patients with medical bills. The hospital system cuts off patients only if they have racked up at least $1,500 of unpaid debt three separate times. It contacts them by phone and with repeated letters that include information about applying for financial help, said Conny Bergerson, a hospital spokeswoman.

“Allina Health’s goal is, and will always be, to have zero patients go without services for financial reasons,” Ms. Bergerson said. She said that cutting off services was “rare” but declined to provide information on how often it happens.

Allina suspended its policy of cutting off patients in March 2020, at the onset of the coronavirus pandemic, before reinstating it in April 2021.

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“Nobody wins when patients can’t get preventive care,” said Dr. Rita Raverty, a primary care doctor who works at an Allina clinic.Credit...Tim Gruber for The New York Times

An estimated 100 million Americans have medical debts. Their bills make up about half of all outstanding debt in the country.

About 20 percent of hospitals nationwide have debt-collection policies that allow them to cancel care, according to an investigation last year by KFF Health News. Many of those are nonprofits. The government does not track how often hospitals withhold care.

Under federal law, hospitals are required to treat everyone who comes to the emergency room, regardless of their ability to pay. But the law — called the Emergency Medical Treatment and Labor Act — is silent on how health systems should treat patients who need other kinds of lifesaving care, like those with aggressive cancers or diabetes.

In 2020, thanks to its nonprofit status, Allina avoided roughly $266 million in state, local and federal taxes, according to the Lown Institute, a think tank that studies health care.

In exchange, the Internal Revenue Service requires Allina and thousands of other nonprofit hospital systems to benefit their local communities, including by providing free or reduced-cost care to patients with low incomes.

But the federal rules do not dictate how poor a patient needs to be to qualify for free care. In 2020, Allina spent less than half of 1 percent of its expenses on charity care, well below the nationwide average of about 2 percent for nonprofit hospitals, according to an analysis of hospital financial filings by Ge Bai, a professor at the Johns Hopkins Bloomberg School of Public Health.

Allina is one of Minnesota’s largest health systems, having largely grown through acquisitions. Since 2013, its annual profits have ranged from $30 million to $380 million. Last year was the first in the past decade when it lost money, largely owing to investment losses.

The financial success has paid dividends. Allina’s president earned $3.5 million in 2021, the most recent year for which data is available. The health system recently built a $12 million conference center.

Yet Allina sometimes plays hardball with patients. Doctors have become accustomed to seeing messages in the electronic medical record notifying them that a patient “will no longer be eligible to receive care” because of “unpaid medical balances.”

Dr. Rita Raverty, a primary care doctor who works at an Allina clinic, said the notifications were alarming because they meant she could not provide continuous care for some of her patients facing a number of health risks.

“Nobody wins when patients can’t get preventive care,” Dr. Raverty said. “It creates worse disease outcomes when you’re not catching things early.”

Doctors and patients described being unable to complete medical forms that children needed to enroll in day care or show proof of vaccination for school.

Serena Gragert, who worked as a scheduler at an Allina clinic in Minneapolis until 2021, said the computer system simply wouldn’t let her book future appointments for some patients with outstanding balances.

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Serena Gragert worked as an appointment scheduler at an Allina clinic in Minneapolis. The scheduling system blocked her from booking patients who had been flagged for unpaid balances.Credit...Tim Gruber for The New York Times

Ms. Gragert and other Allina employees said some of the patients who were kicked out had incomes low enough to qualify for Medicaid, the federal-state insurance program for poor people. That also means those patients would be eligible for free care under Allina’s own financial assistance policy — something many patients are unaware exists when they seek treatment.

Ms. Bergerson, the Allina spokeswoman, did not dispute that but said the health system goes “to tremendous lengths to assist patients with their financial obligations for medical care.”

Allina employees said the policy has forced them to ration care.

Beth Gunhus, a pediatric nurse practitioner, recalled a case in which a mother brought in her three children. One had scabies, an intensely itchy skin condition caused by mites burrowing into the body. She wanted to follow best practices and treat the entire family, who were sharing one bed in a single room they rented, to ensure it didn’t spread further. But she could write a prescription for only two of the children. The third’s account was locked because of unpaid bills.

“There are so many better ways of saving money than what we’re doing,” Ms. Gunhus said.

Allina says the policy applies only to debts related to care provided by its clinics, not its hospitals. But patients said in interviews that they got cut off after falling into debt for services they received at Allina’s hospitals.

Because Allina is the dominant health system in some rural parts of Minnesota, getting kicked out can leave patients with few options.

Jennifer Blaido lives in Isanti, a small town outside Minneapolis, and Allina owns the only hospital there. Ms. Blaido, a mechanic, said she racked up nearly $200,000 in bills from a two-week stay at Allina’s Mercy Hospital in 2009 for complications from pneumonia, along with several visits to the emergency department for asthma flare-ups. Ms. Blaido, a mother of four, said most of the hospital stay was not covered by her health insurance and she was unable to scrounge together enough money to make a dent in the debt.

Last year, Ms. Blaido had a cancer scare and said she couldn’t get an appointment with a doctor at Mercy Hospital. She had to drive more than an hour to get examined at a health system unconnected to Allina.

Allina does not make this policy explicit to patients. It is not mentioned in the health system’s list of “frequently asked questions” about billing practices. In at least one case, Allina has denied that it even existed.

In a lawsuit filed last year in state court in Minnesota, Allina sued a couple, Jordan and JoLynda Anderson, for nearly $10,000 in unpaid medical bills.

In court filings, the couple described how Allina canceled Ms. Anderson’s appointments and told her that she could not book new ones until she had set up three separate payment plans — one with the health system and two with its debt collectors.

Even after setting up those payment plans, which totaled $580 a month, the canceled appointments were never restored. Allina allows patients to come back only after they have paid the entire debt.

Ms. Anderson recalls being devastated about losing her visit to an endocrinologist that specialized in a chronic condition she has. She had already been waiting four months for the appointment, and was unable to get a new one.

“It felt like I was being punished, and the punishment was you get to stay ill,” she said.

Ms. Bergerson declined to comment on these cases, citing patient privacy.

When the Andersons asked in court for a copy of Allina’s policy of barring patients with unpaid bills, the hospital’s lawyers responded: “Allina does not have a written policy regarding the canceling of services or termination of scheduled and/or physician referral services or appointments for unpaid debts.”

In fact, Allina’s policy, which was created in 2006, instructs employees on how to do exactly that. Among other things, it tells staff to “cancel any future appointments the patient has scheduled at any clinic.”

It does provide a few ways for patients to continue being seen despite their unpaid bills. One is by getting approved for a loan through the hospital. Another is by filing for bankruptcy.

https://www.nytimes.com/2023/06/01/business/allina-health-hospital-debt.html

Novan Focuses Strategic Direction and Announces Restructuring

  Company directing resources on the potential approval of berdazimer gel, 10.3% (SB206) for molluscum contagiosum –

– Process to explore a sale or out-license of commercial assets or other business transaction continues –

– Continued progress toward PDUFA goal date of January 5, 2024, for berdazimer gel, 10.3% –

Novan, Inc. (“the Company” or “Novan”) (Nasdaq: NOVN), today announced that following an evaluation of its commercial and developmental stage assets, it has decided to sharpen its focus and resources on berdazimer gel, 10.3% (SB206) and announced that it continues to explore strategic alternatives, with a focus on its commercial product portfolio and a sale or out-license of one or more of its commercial products.

Novan conducted a deliberate and thorough review of its commercial and development portfolio of assets to determine a path to optimally deploy capital and maximize shareholder value. Following this review, the Company, has initiated a process to explore a sale or out-license of its commercial assets, including RHOFADE, MINOLIRA, and CLODERM. The Company will immediately reduce certain of its operating expenses currently supporting its commercial operations.

“We continue to face serious challenges achieving profitability with our commercial assets in the current economic environment, while also endeavoring to extend our cash runway through the PDUFA goal date of January 5, 2024, for berdazimer gel, 10.3%,” said Paula Brown Stafford, President and CEO. “We have been evaluating our portfolio of assets for some time to determine the optimal path for a product, if approved, to treat molluscum contagiosum, an unmet medical need. After careful consideration, we have made the strategic decision to implement a reduction in force of our commercial team and continue to explore a sale or out-license of our commercial assets. We will use our resources, plus the proceeds from any dilutive or non-dilutive financing or any strategic transaction, to work in earnest to capture the potential value of berdazimer gel, 10.3% by supporting the ongoing NDA review process.”

https://finance.yahoo.com/news/novan-focuses-strategic-direction-announces-200500012.html