Peter Marks, CBER director (Greg Nash/Pool via AP Images)

Eye in­jec­tions caus­ing blind­ness? FDA’s stem cell clin­ic crack­down read­ies for ac­tion

Be­gin­ning next week, the FDA will of­fi­cial­ly start a long-await­ed crack­down on stem cell clin­ics hawk­ing un­proven treat­ments that have no in­ten­tion of go­ing through the of­fi­cial process of test­ing their prod­ucts in clin­i­cal tri­als.

More than 1,000 of these di­rect-to-con­sumer stem cell clin­ics have popped up across the coun­try in re­cent years, promis­ing help for con­di­tions with lim­it­ed to no treat­ment op­tions, like Alzheimer’s, ALS, autism or even Covid-19, and no clin­i­cal ev­i­dence to back their pricey prod­ucts.

“Ob­vi­ous­ly, it can’t be that on June 1 we’re go­ing to shut every­thing down, be­cause we don’t have the band­width,” FDA’s top bi­o­log­ics head Pe­ter Marks told End­points News in an in­ter­view. “But we’re go­ing to make very clear that we are se­ri­ous about putting an end to prod­ucts that should be un­der in­ves­ti­ga­tion­al new drug ap­pli­ca­tions or bi­o­log­ic li­cense ap­pli­ca­tions.”

The FDA has pro­vid­ed these clin­ics with plen­ty of lee­way in the in­ter­ven­ing years (al­though any safe­ty events must be re­port­ed to the agency by law) — of­fer­ing more than 3 years of en­force­ment dis­cre­tion while pro­vid­ing rapid re­spons­es to any in­quiries from stem clin­ics that want­ed to get up to speed on what they need to sub­mit to the FDA.

“Peo­ple de­serve to have med­ical prod­ucts that work. It’s quite an irony that peo­ple who are pay­ing out of pock­et are of­ten get­ting prod­ucts that have ze­ro ef­fi­ca­cy or even worse, they could be harm­ful,” Marks added.

In­deed, the FDA has doc­u­ment­ed such se­ri­ous ad­verse events over the years, in­clud­ing at least one pa­tient be­com­ing blind due to an in­jec­tion of stem cells in­to the eye, and an­oth­er re­ceiv­ing a spinal cord in­jec­tion that caused the growth of a tu­mor.

Back in 2017, US Mar­shals, at the re­quest of FDA, al­so seized five vials of a small­pox vac­cine con­tain­ing live cow pox virus that were dis­cov­ered dur­ing an in­spec­tion of a San Diego-based stem cell clin­ic pur­port­ing to spe­cial­ize in “im­mune-on­col­o­gy.”

“Even if stem cells are your own cells, there are still safe­ty risks,” FDA ex­plains, not­ing that the ma­nip­u­la­tion of such cells af­ter re­moval can lead to con­t­a­m­i­na­tion.

And while the FDA can con­tin­ue to is­sue warn­ings and un­ti­tled let­ters, as it has over the years, Marks al­so made clear that the agency can take tougher ac­tion to close busi­ness­es that are act­ing egre­gious­ly.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,000+ biopharma pros reading Endpoints daily — and it's free.

Jeffrey Nau, Oyster Point Pharma CEO

FDA OKs an in­haled ver­sion of smok­ing ces­sa­tion drug Chan­tix — for a com­mon eye dis­ease

Oyster Point Pharma now has its first FDA-approved product — Tyrvaya. And the biotech has taken a unique route to get there by using an old drug with a storied past.

The New Jersey biotech announced this morning that the FDA has approved their nasal spray product for dry eye disease on Friday — the first nasal spray to be approved for the disease. The product’s active ingredient is 0.03 mg of varenicline, also known as smoking cessation aid Chantix.

Omeros plunges deep­er af­ter FDA re­jects rare dis­ease drug, ask­ing for more in­for­ma­tion

Omeros practically warned investors that a complete response letter was coming when it disclosed that the FDA found deficiencies in its BLA for narsoplimab. But the agency did not elaborate on the specifics of those deficiencies for the drug, which was being positioned as a treatment for the rare but serious blood clotting disease known as hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).

UP­DAT­ED: Third time's the charm: Adamis nabs ap­proval of high-dose nalox­one in­jec­tion af­ter two CRLs

If at first you don’t succeed, and at second you don’t succeed, try, try again.

That’s been the unofficial mantra for Adamis Pharmaceuticals’ high dose naloxone injection, which received two CRLs in the span of a year between the Novembers of 2019 and 2020. But on Monday, word came through that the FDA approved the drug on Adamis’ third attempt, giving doctors another tool to treat individuals who overdose on opioids.

Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,000+ biopharma pros reading Endpoints daily — and it's free.

Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,000+ biopharma pros reading Endpoints daily — and it's free.

Mark Foley, Revance CEO (Revance via Vimeo)

UP­DAT­ED: FDA re­jects Re­vance's pitch for Botox com­peti­tor as biotech blames man­u­fac­tur­ing is­sues

The FDA has told Revance Therapeutics that, no, it won’t be able to turn those frowns upside down.

Regulators issued a CRL to the biotech for its botox-based therapy for moderate to severe glabellar lines, also known as frown lines, Revance announced Friday. As is agency policy, the FDA does not comment on these types of communications, but Revance said the rejection came from deficiencies at a manufacturing site.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,000+ biopharma pros reading Endpoints daily — and it's free.